Successful Peripheral T-Lymphocyte–Directed Gene Transfer for a Patient With Severe Combined Immune Deficiency Caused by Adenosine Deaminase Deficiency

Autor: Kotani H, Masafumi Yamada, Ichiro Kobayashi, Shuzo Matsumoto, McGarrity Gj, Makoto Ohtsu, Tadashi Ariga, Motohiko Okano, M. Onodera, Atsushi Tame, Hirofumi Furuta, Nobuaki Kawamura, Blaese Rm, Yukio Sakiyama

Publikováno v: Blood. 91:30-36

Ten patients with adenosine deaminase deficiency (ADA−) have been enrolled in gene therapy clinical trials since the first patient was treated in September 1990. We describe a Japanese ADA− severe combined immune deficiency (SCID) patient who has

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7ff09b96fc3721648c9baf0f019c7146
https://doi.org/10.1182/blood.v91.1.30

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It's time to end RAC review of gene therapy protocols.

Autor: McGarrity GJ

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2012 Jun; Vol. 20 (6), pp. 1079-80.

Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery.

Autor: Zayed H; University of Minnesota Cancer Center, Department of Pediatrics, Division of Hematology-Oncology, Blood and Marrow Transplantation, University of Minnesota, Minneapolis, USA., Xia L, Yerich A, Yant SR, Kay MA, Puttaraju M, McGarrity GJ, Wiest DL, McIvor RS, Tolar J, Blazar BR

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2007 Jul; Vol. 15 (7), pp. 1273-9. Date of Electronic Publication: 2007 Apr 24.