Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome

Autor: Magnani, A., Semeraro, M., Adam, F., Booth, C., Dupré, L., Morris, E. C., Gabrion, A., Roudaut, C., Borgel, D., Toubert, A., Clave, E., Abdo, C., Gorochov, G., Petermann, R., Guiot, M., Miyara, M., Moshous, D., Magrin, E., Denis, A., Suarez, F., Lagresle, C., Roche, A. M., Everett, J., Trinquand, A., Guisset, M., Bayford, J. Xu, Hacein-Bey-Abina, S., Kauskot, A., Elfeky, R., Rivat, C., Abbas, S., Gaspar, H. B., Macintyre, E., Picard, C., Bushman, F. D., Galy, A., Fischer, A., Six, E., Thrasher, A. J., Cavazzana, M.

Publikováno v: NATURE MEDICINE
r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
Universidad de las Islas Baleares
Nature Medicine
Nature Medicine, 2022, 28 (1), pp.71-80. ⟨10.1038/s41591-021-01641-x⟩

Patients with Wiskott–Aldrich syndrome (WAS) lacking a human leukocyte antigen-matched donor may benefit from gene therapy through the provision of gene-corrected, autologous hematopoietic stem/progenitor cells. Here, we present comprehensive, long

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5f3f98b89d4b68a773ab2a2e9643726a
http://fundanet.fsjd.org/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=21058

Therapeutic Benefit and Gene Network Regulation by Combined Gene Transfer of Apelin, FGF2, and SERCA2a into Ischemic Heart

Autor: Philippe Valet, Benoit Lebas, Bernard Masri, Pierre Sicard, Barbara Garmy-Susini, Jerome Roncalli, Fransky Hantelys, Françoise Pujol, Oksana Kunduzova, Anne-Catherine Prats, Xavier Chaufour, Edith Renaud-Gabardos, Angelo Parini, Florence Tatin, Denis Calise

Publikováno v: Molecular Therapy
Molecular Therapy, Nature Publishing Group, 2018, 26 (3), pp.902-916. ⟨10.1016/j.ymthe.2017.11.007⟩
Molecular Therapy, Cell Press, 2018, 26 (3), pp.902-916. ⟨10.1016/j.ymthe.2017.11.007⟩
Molecular Therapy, 2018, 26 (3), pp.902-916. ⟨10.1016/j.ymthe.2017.11.007⟩

International audience; Despite considerable advances in cardiovascular disease treatment, heart failure remains a public health challenge. In this context, gene therapy appears as an attractive approach, but clinical trials using single therapeutic

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::eff05544b5df3c872bf90d9754a0bbb3
https://hal-univ-evry.archives-ouvertes.fr/hal-02393129

Modeling amyotrophic lateral sclerosis in pure human iPSc-derived motor neurons isolated by a novel FACS double selection technique

Autor: Dorothée Buttigieg, Stéphane Blanchard, Diana Toli, Delphine Bohl, Georg Haase, Gilbert Baillat, Sarah Bellouze, Thomas Lemonnier, Boris Lamotte d'Incamps

Publikováno v: Neurobiology of Disease, Vol 82, Iss, Pp 269-280 (2015)
Neurobiology of Disease
Neurobiology of Disease, Elsevier, 2015, 82, pp.269-280. ⟨10.1016/j.nbd.2015.06.011⟩
Neurobiology of Disease, 2015, 82, pp.269-280. ⟨10.1016/j.nbd.2015.06.011⟩

International audience; Amyotrophic lateral sclerosis (ALS) is a severe and incurable neurodegenerative disease. Human motor neurons generated from induced pluripotent stem cells (iPSc) offer new perspectives for disease modeling and drug testing in

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5c65ffa44a284ec8da4026ffb27e4be6
https://doi.org/10.1016/j.nbd.2015.06.011

Lentiviral-mediated gene transfer of brain-derived neurotrophic factor is neuroprotective in a mouse model of neonatal excitotoxic challenge

Autor: Pierre Gressens, Isabelle Husson, Muriel Jaquet, Jacques Mallet, Barry E. Kosofsky, Alexis-Pierre Bemelmans

Publikováno v: Journal of Neuroscience Research
Journal of Neuroscience Research, 2006, 83 (1), pp.50-60. ⟨10.1002/jnr.20704⟩
Journal of Neuroscience Research, Wiley, 2006, 83 (1), pp.50-60. ⟨10.1002/jnr.20704⟩

Excitotoxicity may be a critical factor in the formation of brain lesions associated with cerebral palsy. When injected into the murine neopallium at postnatal day 5, the glutamatergic analog N-methyl-D-aspartate (NMDA) produces transcortical neurona

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::96df00671c709ea24f6cbd2d8d7ffae0
https://doi.org/10.1002/jnr.20704

Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells

Autor: Pierre Charneau, Julia Hauer, Julie Rivière, Marc Le Lorc'h, Jean-Pierre de Villartay, Pauline Soulas-Sprauel, Anne Galy, Michèle Malassis-Séris, Johanna Blondeau, Alexandrine Garrigue, Alain Fischer, Frederique Pâques, Nicole Brousse, Marina Cavazzana-Calvo, Serge Romana, Fatine Benjelloun, Daniel Stockholm, Annick Lim, Corinne Demerens-de Chappedelaine

Publikováno v: Molecular Therapy
Molecular Therapy, Cell Press, 2008, 16 (8), pp.1490-9. ⟨10.1038/mt.2008.118⟩
Molecular Therapy, 2008, 16 (8), pp.1490-1499. ⟨10.1038/mt.2008.118⟩
Molecular Therapy, Nature Publishing Group, 2008, 16 (8), pp.1490-9. 〈10.1038/mt.2008.118〉
Molecular Therapy, 2008, 16 (8), pp.1490-9. ⟨10.1038/mt.2008.118⟩
Molecular Therapy, Nature Publishing Group, 2008, 16 (8), pp.1490-9. ⟨10.1038/mt.2008.118⟩

International audience; Patients with mutations in the Artemis gene display a complete absence of T- and B lymphocytes, together with increased cellular radiosensitivity; this leads to a radiosensitive severe combined immunodeficiency (RS-SCID). Allo

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::dd124b09ba43175b099dad8fc5b27673
https://pubmed.ncbi.nlm.nih.gov/18560421