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

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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Hlavní autoři: Toru Uchiyama, Sirirat Takahashi, Kazuhiko Nakabayashi, Kohji Okamura, Kaori Edasawa, Masafumi Yamada, Nobuyuki Watanabe, Emi Mochizuki, Toru Yasuda, Akane Miura, Motohiro Kato, Daisuke Tomizawa, Makoto Otsu, Tadashi Ariga, Masafumi Onodera
Médium: Článek
Jazyk:English
Vydáno: Elsevier 2021-12-01
Edice:Molecular Therapy: Methods & Clinical Development
Témata:
ADA-SCID
retroviral vector
nonconditioned gene therapy
clonal dominance
ADA activity
insertional mutagenesis
On-line přístup:http://www.sciencedirect.com/science/article/pii/S2329050121001601

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http://www.sciencedirect.com/science/article/pii/S2329050121001601

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