Zobrazeno 41 - 47
of 47
pro vyhledávání: '"Monpoux, Fabrice"'
Autor:
Sirvent, Nicolas, Monpoux, Fabrice, Benet, Laurent, Richelme, Christian, Mariani, Roger, Diaine, Bernard
Publikováno v:
Medical & Pediatric Oncology; 1998, Vol. 30 Issue 5, p308-308, 1p
Autor:
Chappell, E., Kohns Vasconcelos, M., Goodall, R. L., Galli, L., Goetghebuer, T., Noguera-Julian, A., Rodrigues, L. C., Scherpbier, H., Smit, C., Bamford, A., Crichton, S., Navarro, M. L., Ramos, J. T., Warszawski, J., Spolou, V., Chiappini, E., Venturini, E., Prata, F., Kahlert, C., Marczynska, M., Marques, L., Naver, L., Thorne, C., Gibb, D. M., Giaquinto, C., Judd, A., Collins, I. J., Goodall, R., Rodrigues, L., Duff, C., Gomezpena, D., Jackson, C., Lundin, R., Mangiarini, L., Milanzi, E., Nardone, A., Hainaut, M., Van der Kelen, E., Delforge, M., Le Chenadec, J., Ramos, E., Dialla, O., Wack, T., Laurent, C., Ait si Selmi, L., Leymarie, I., Ait Benali, F., Brossard, M., Boufassa, L., Floch-Tudal, C., Firtion, G., Hau, I., Chace, A., Bolot, P., Blanche, S., Granier, M., Labrune, P., Lachassine, E., Dollfus, C., Levine, M., Fourcade, C., Heller-Roussin, B., Runel-Belliard, C., Tricoire, J., Monpoux, F., Chirouze, C., Reliquet, V., Brouard, J., Kebaili, K., Fialaire, P., de Villeneuve, A., Lalande, M., de Flandres, J., Mazingue, F., Partisani, M. L., de Martino, M., Angelo Tovo, P., Gabiano, C., Carloni, I., Larovere, D., Baldi, F., Miniaci, A., Pession, A., Badolato, R., Panto, G., Anastasio, E., Montagnani, C., Bianchi, L., Allodi, A., Di Biagio, A., Grignolo, S., Giacomet, V., Marchisio, P., Banderali, G., Tagliabue, C., Cellini, M., Bruzzese, E., Di Costanzo, P., Lo Vecchio, A., Dona, D., Rampon, O., Romano, A., Dodi, I., Esposito, S., Zuccaro, V., Zanaboni, D., Consolini, R., Bernardi, S., Genovese, O., Cristiano, L., Mazza, A., Garazzino, S., Mignone, F., Silvestro, E., Portelli, V., Kinderziekenhuis, E., van der Kuip, M., Pajkrt, D., Scherpbier, H. J., de Boer, C., Weijsenfeld, A. M., Jurriaans, S., Back, N. K. T., Zaaijer, H. L., Berkhout, B., Cornelissen, M. T. E., Schinkel, C. J., Wolthers, K. C., Fraaij, P. L. A., van Rossum, A. M. C., Vermont, C. L., van der Knaap, L. C., Visser, E., Boucher, C. A. B., Koopmans, M. P. G., van Kampen, J. J. A., Henriet, S. S. V., van Aerde, M. K., Strik-Albers, R., Rahamat-Langendoen, J., Stelma, F. F., Burger, D., Scholvinck, E. H., de Groot-de Jonge, H., Niesters, H. G. M., van Leer-Buter, C. C., Knoester, M., Bont, L. J., Geelen, S. P. M., Loeffen, Y. G. T., Wolfs, T. F. W., Nauta, N., Schuurman, R., Hofstra, L. M., Wensing, A. M. J., Reiss, P., Zaheri, S., Boyd, A. C., Bezemer, D. O., van Sighem, A. I., Wit, F. W. M. N., Hillebregt, M. M. J., Woudstra, T. J., Bergsma, D., van de Sande, L., Rutkens, T., van der Vliet, S., Lelivelt, K. J., Scheijgrond, A., de Groot, L., van den Akker, M., Bakker, Y., EI Berkaoui, A., Bezemer, M., Bretin, N., Djoechro, E., Groters, M., Kruijne, E., Lodewijk, C., Lucas, E., Munjishvili, L., Paling, F., Peeck, B., Ree, C., Regtop, R., Ruijs, Y., Schoorl, M., Schnorr, P., Tuijn, E., Veenenberg, L., Visser, K. M., Witte, E. C., Popielska, J., Pokorska-Spiewak, M., Oldakowska, A., Zawadka, K., Coupland, U., Doroba, M., Teixeira, C., Fernandes, A., Soler-Palacin, P., Antoinette Frick, M., Perez-Hoyos, S., Mur, A., Lopez, N., Mendez, M., Mayol, L., Vallmanya, T., Calavia, O., Garcia, L., Coll, M., Pineda, V., Rius, N., Rovira, N., Duenas, J., Fortuny, C., Jose Mellado, M., Escosa, L., Garcia Hortelano, M., Sainz, T., Gonzalez-Tome, M. I., Rojo, P., Blazquez, D., Prieto-Tato, L., Epalza, C., Tomas Ramos, J., Guillen, S., Saavedra, J., Santos, M., Santiago, B., de Ory, S. J., Carrasco, I., Munoz-Fernandez, M. A., Angel Roa, M., Penin, M., Martinez, J., Badillo, K., Onate, E., Pocheville, I., Garrote, E., Colino, E., Gomez Sirvent, J., Garzon, M., Roman, V., Angulo, R., Neth, O., Falcon, L., Terol, P., Luis Santos, J., Moreno, D., Lendinez, F., Peromingo, E., Uberos, J., Ruiz, B., Grande, A., Jose Romero, F., Perez, C., Lillo, M., Losada, B., Herranz, M., Bustillo, M., Collado, P., Antonio Couceiro, J., Vila, L., Calvino, C., Isabel Piqueras, A., Oltra, M., Gavilan, C., Montesinos, E., Dapena, M., Alvarez, C., Jimenez, B., Gloria Andres, A., Marugan, V., Ochoa, C., Alfayate, S., Isabel Menasalvas, A., del Prado, Y. R., Navernaver, L., Soeria-Atmadja, S., Belfrage, E., Hagas, V., Aebi-Popp, K., Anagnostopoulos, A., Battegay, M., Baumann, M., Bernasconi, E., Boni, J., Braun, D. L., Bucher, H. C., Calmy, A., Cavassini, M., Ciuffi, A., Crisinel, P. A., Duppenthaler, A., Dollenmaier, G., Egger, M., Elzi, L., Fehr, J., Fellay, J., Francini, K., Furrer, H., Fux, C. A., Gunthard, H. F., Haerry, D., Hasse, B., Hirsch, H. H., Hoffmann, M., Hosli, I., Huber, M., Kaiser, L., Keiser, O., Klimkait, T., Kottanattu, L., Kouyos, R. D., Kovari, H., Ledergerber, B., Martinetti, G., Martinez de Tejada, B., Marzolini, C., Metzner, K. J., Muller, N., Nicca, D., Paioni, P., Pantaleo, G., Perreau, M., Polli, C., Rauch, A., Rudin, C., Scherrer, A. U., Schmid, P., Speck, R., Stockle, M., Sultan-Beyer, L., Tarr, P., Thanh Lecompte, M., Trkola, A., Vernazza, P., Wagner, N., Wandeler, G., Weber, R., Yerly, S., Lyall, H., Butler, K., Doerholt, K., Doherty, C., Foster, C., Harrison, I., Kenny, J., Klein, N., Letting, G., Mcmaster, P., Murau, F., Nsangi, E., Prime, K., Riordan, A., Shackley, F., Shingadia, D., Storey, S., Tudor-Williams, G., Turkova, A., Welch, S., Cook, C., Dobson, D., Fairbrother, K., Prevost, M. L., Van Looy, N., Peters, H., Francis, K., Thrasyvoulou, L., Fidler, K., Bernatoniene, J., Manyika, F., Sharpe, G., Subramaniam, B., Hague, R., Price, V., Flynn, J., Cardoso, A., Abou - Rayyah, M., Yeadon, S., Segal, S., Hawkins, S., Dowie, M., Bandi, S., Percival, E., Eisenhut, M., Duncan, K., Anguvaa, L., Wren, L., Flood, T., Pickering, A., Murphy, C., Daniels, J., Lees, Y., Thompson, F., Williams, A., Williams, B., Pope, S., Libeschutz, S., Cliffe, L., Southall, S., Freeman, A., Freeman, H., Christie, S., Gordon, A., Rosie Hague, D., Clarke, L., Jones, L., Brown, L., Greenberg, M., Benson, C., Ibberson, L., Patel, S., Hancock, J., Sharland, M., Lyall, E. G. H., Seery, P., Kirkhope, N., Raghunanan, S., Callaghan, A., Bridgwood, A., Evans, J., Blake, E., Yannoulias, A.
Publikováno v:
HIV Medicine
HIV Medicine, Wiley, 2021, ⟨10.1111/hiv.13177⟩
HIV medicine. Wiley-Blackwell
Hiv Medicine, 23, 2, pp. 186-196
Hiv Medicine, 23, 186-196
van der Kuip, M 2021, ' Children living with HIV in Europe: do migrants have worse treatment outcomes? ', HIV Medicine . https://doi.org/10.1111/hiv.13177
HIV MEDICINE
r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
instname
HIV Medicine, 23(2), 186-196. Wiley-Blackwell Publishing Ltd
HIV Medicine, Wiley, 2021, ⟨10.1111/hiv.13177⟩
HIV medicine. Wiley-Blackwell
Hiv Medicine, 23, 2, pp. 186-196
Hiv Medicine, 23, 186-196
van der Kuip, M 2021, ' Children living with HIV in Europe: do migrants have worse treatment outcomes? ', HIV Medicine . https://doi.org/10.1111/hiv.13177
HIV MEDICINE
r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
instname
HIV Medicine, 23(2), 186-196. Wiley-Blackwell Publishing Ltd
Contains fulltext : 249078.pdf (Publisher’s version ) (Open Access) OBJECTIVES: To assess the effect of migrant status on treatment outcomes among children living with HIV in Europe. METHODS: Children aged
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a95d753ffd01848240447cbb0ec01ddd
http://hdl.handle.net/2318/1870163
http://hdl.handle.net/2318/1870163
Autor:
Raynaud, Sophie D., Brunet, Benedicte, Ayraud, Noel, Monpoux, Fabrice, Philip, Patrick, Bayle, Jacques
Publikováno v:
In Cancer Genetics and Cytogenetics 1993 71(1):100-101
Autor:
Cerato-Blanc C; Service d'hémato-oncologie infantile, hôpital Archet 2, centre hospitalier universitaire de Nice, 151 route Saint-Antoine-de-Ginestière, 06202 Nice, France. Electronic address: cerato-blanc.c@chu-nice.fr., Monpoux F; Service d'hémato-oncologie infantile, hôpital Archet 2, centre hospitalier universitaire de Nice, 151 route Saint-Antoine-de-Ginestière, 06202 Nice, France.
Publikováno v:
Revue de l'infirmiere [Rev Infirm] 2021 Oct; Vol. 70 (274), pp. 42-44. Date of Electronic Publication: 2021 Jul 19.
Autor:
Thompson AA; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Walters MC; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Kwiatkowski J; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Rasko JEJ; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Ribeil JA; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Hongeng S; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Magrin E; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Schiller GJ; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Payen E; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Semeraro M; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Moshous D; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Lefrere F; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Puy H; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Bourget P; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Magnani A; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Caccavelli L; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Diana JS; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Suarez F; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Monpoux F; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Brousse V; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Poirot C; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Brouzes C; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Meritet JF; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Pondarré C; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Beuzard Y; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Chrétien S; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Lefebvre T; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Teachey DT; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Anurathapan U; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Ho PJ; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., von Kalle C; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Kletzel M; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Vichinsky E; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Soni S; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Veres G; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Negre O; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Ross RW; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Davidson D; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Petrusich A; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Sandler L; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Asmal M; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Hermine O; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., De Montalembert M; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Hacein-Bey-Abina S; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Blanche S; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Leboulch P; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.)., Cavazzana M; From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.).
Publikováno v:
The New England journal of medicine [N Engl J Med] 2018 Apr 19; Vol. 378 (16), pp. 1479-1493.
Autor:
Monpoux F; Service de Pédiatrie, Hôpital de l'Archet II, 151, Route de Saint Antoine de Ginestière, BP 3079 - 06202 Nice Cedex 3, France. monpoux.f@chu-nice.fr, Tricoire J, Lalande M, Reliquet V, Bebin B, Thuret I
Publikováno v:
AIDS (London, England) [AIDS] 2004 Dec 03; Vol. 18 (18), pp. 2401-9.