Nonconditioned ADA-SCID gene therapy reveals ADA requirement in the hematopoietic system and clonal dominance of vector-marked clones

Two patients with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (ADA-SCID) received stem cell-based gene therapy (SCGT) using GCsapM-ADA retroviral vectors without preconditioning in 2003 and 2004. The first patient (Pt1) was treated at 4.7 years old, and the second patient (P...

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Detalles Bibliográficos
Autores principales: Uchiyama, Toru, Takahashi, Sirirat, Nakabayashi, Kazuhiko, Okamura, Kohji, Edasawa, Kaori, Yamada, Masafumi, Watanabe, Nobuyuki, Mochizuki, Emi, Yasuda, Toru, Miura, Akane, Kato, Motohiro, Tomizawa, Daisuke, Otsu, Makoto, Ariga, Tadashi, Onodera, Masafumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8566957/
https://www.ncbi.nlm.nih.gov/pubmed/34786435
http://dx.doi.org/10.1016/j.omtm.2021.10.003

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8566957/
https://www.ncbi.nlm.nih.gov/pubmed/34786435
http://dx.doi.org/10.1016/j.omtm.2021.10.003

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