Zobrazeno 1 - 10
of 260
pro vyhledávání: '"GT, Gene therapy"'
Autor:
Pete Lollar
Publikováno v:
The Hematologist. 9
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::d6f6843cccadfff68a1d5dd6e6f54c7c
https://doi.org/10.1182/hem.v9.2.1180
https://doi.org/10.1182/hem.v9.2.1180
Autor:
Marco Spinelli, Chiara Brombin, Nufar Marcus, Dario Di Silvestre, Patrizia Della Valle, Lucia Piceni Sereni, Maria Ester Bernardo, Pierluigi Mauri, Francesca Ferrua, Claudio Pignata, Lorella Cattaneo, Alessandro Aiuti, Lucia Dora Notarangelo, Armando D'Angelo, Marita Bosticardo, Stefania Giannelli, Koen van Rossem, Maddalena Migliavacca, Anna Villa, Loris Pozzi, Roula Farah, Maria Carmina Castiello, Maria Pia Cicalese
Publikováno v:
Journal of allergy and clinical immunology 144 (2019): 825–838. doi:10.1016/j.jaci.2019.03.012
info:cnr-pdr/source/autori:Sereni L1, Castiello MC1, Di Silvestre D2, Della Valle P3, Brombin C4, Ferrua F5, Cicalese MP6, Pozzi L3, Migliavacca M6, Bernardo ME6, Pignata C7, Farah R8, Notarangelo LD9, Marcus N10, Cattaneo L11, Spinelli M12, Giannelli S1, Bosticardo M1, van Rossem K13, D'Angelo A3, Aiuti A5, Mauri P2, Villa A14./titolo:Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome/doi:10.1016%2Fj.jaci.2019.03.012/rivista:Journal of allergy and clinical immunology/anno:2019/pagina_da:825/pagina_a:838/intervallo_pagine:825–838/volume:144
The Journal of Allergy and Clinical Immunology
info:cnr-pdr/source/autori:Sereni L1, Castiello MC1, Di Silvestre D2, Della Valle P3, Brombin C4, Ferrua F5, Cicalese MP6, Pozzi L3, Migliavacca M6, Bernardo ME6, Pignata C7, Farah R8, Notarangelo LD9, Marcus N10, Cattaneo L11, Spinelli M12, Giannelli S1, Bosticardo M1, van Rossem K13, D'Angelo A3, Aiuti A5, Mauri P2, Villa A14./titolo:Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome/doi:10.1016%2Fj.jaci.2019.03.012/rivista:Journal of allergy and clinical immunology/anno:2019/pagina_da:825/pagina_a:838/intervallo_pagine:825–838/volume:144
The Journal of Allergy and Clinical Immunology
BACKGROUND: Thrombocytopenia is a serious issue for all patients with classical Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia (XLT) because it causes severe and life-threatening bleeding. Lentiviral gene therapy (GT) for WAS has shown
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8fbef2a3b9359481a3aee0ecf5da45bb
https://publications.cnr.it/doc/417797
https://publications.cnr.it/doc/417797
Autor:
Barrois, R.1,2,3,4 (AUTHOR) remi.barrois@aphp.fr, Tervil, B.3 (AUTHOR), Cacioppo, M.5,6 (AUTHOR), Barnerias, C.1 (AUTHOR), Deladrière, E.1 (AUTHOR), Leloup-Germa, V.1 (AUTHOR), Hervé, A.1 (AUTHOR), Oudre, L.3 (AUTHOR), Ricard, D.3,7 (AUTHOR), Vidal, P. P.3 (AUTHOR), Vayatis, N.3 (AUTHOR), Roy, S. Quijano8 (AUTHOR), Brochard, S.6,9 (AUTHOR), Gitiaux, C.1,2 (AUTHOR), Desguerre, I.1,10 (AUTHOR)
Publikováno v:
Journal of NeuroEngineering & Rehabilitation (JNER). 10/17/2024, Vol. 21 Issue 1, p1-16. 16p.
Autor:
Corazolla, Eleonore M.1,2,3 (AUTHOR), Eskes, Eline C. B.1,3 (AUTHOR), Veldwijk, Jorien4,5 (AUTHOR), Brands, Marion M. M. G.3,6 (AUTHOR), Dekker, Hanka7 (AUTHOR), van de Mheen, Erica8 (AUTHOR), Langeveld, Mirjam1,3 (AUTHOR), Hollak, Carla E. M.1,3 (AUTHOR), Sjouke, Barbara3,9 (AUTHOR) barbara.sjouke@radboudumc.nl
Publikováno v:
Orphanet Journal of Rare Diseases. 10/3/2024, Vol. 19 Issue 1, p1-16. 16p.
Autor:
Paolo Uva, Mirjam van der Burg, Anna Villa, Immacolata Brigida, Francesca Ferrua, Lucia Piceni Sereni, Michael H. Albert, Giorgio Ottaviano, Marita Bosticardo, Alessandro Aiuti, Maria Carmina Castiello, Francesca Pala, Maria Grazia Roncarolo, Luigi Naldini, Samantha Scaramuzza
Publikováno v:
Journal of Allergy and Clinical Immunology, 136(3), 692-+. Mosby Inc.
The Journal of Allergy and Clinical Immunology
The Journal of Allergy and Clinical Immunology
Background Wiskott-Aldrich syndrome (WAS) is a severe X-linked immunodeficiency characterized by microthrombocytopenia, eczema, recurrent infections, and susceptibility to autoimmunity and lymphomas. Hematopoietic stem cell transplantation is the tre
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::495ffab946a30ddf28166ff0f448fa5a
https://doi.org/10.1016/j.jaci.2015.01.035
https://doi.org/10.1016/j.jaci.2015.01.035
Autor:
Lazea, Cecilia1,2 (AUTHOR) cecilialazea@umfcluj.ro, Vulturar, Romana3,4,5 (AUTHOR) ldamian.reumatologie@gmail.com, Chiș, Adina3,4,5 (AUTHOR) adinachis82@gmail.com, Encica, Svetlana6 (AUTHOR) s_encica@yahoo.com, Horvat, Melinda7 (AUTHOR) melinda.horvat@umfcluj.ro, Belizna, Cristina8,9 (AUTHOR) cristina.belizna@wanadoo.fr, Damian, Laura-Otilia5,10,11 (AUTHOR)
Publikováno v:
International Journal of Molecular Sciences. May2024, Vol. 25 Issue 10, p5567. 33p.
Publikováno v:
Perspectives in Clinical Research. Jan-Mar2024, Vol. 15 Issue 1, p4-9. 6p.
Autor:
Aerts-Kaya, Fatima1,2 (AUTHOR) fatima.aerts@hacettepe.edu.tr, van Til, Niek P.3,4 (AUTHOR) n.p.vantil@amsterdamumc.nl
Publikováno v:
Pharmaceutics. Nov2023, Vol. 15 Issue 11, p2522. 25p.
Autor:
Wenqing Liang, Chao Zhou, Yongjun Deng, Lifeng Fu, Jiayi Zhao, Hengguo Long, Wenyi Ming, Jinxiang Shang, Bin Zeng
Publikováno v:
Annals of Medicine; Dec2024, Vol. 56 Issue 1, p1-22, 22p
Publikováno v:
Pharmeuropa Bio & Scientific Notes; Nov2024, p221-233, 13p