Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease-Reference-Cited by-同舟云学术

Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease

Published:2016-03-07 Issue:4 Volume:34 Page:424-429
ISSN:1087-0156
Container-title:Nature Biotechnology
language:en
Short-container-title:Nat Biotechnol

Author:

De Ravin Suk See,Reik Andreas,Liu Pei-Qi,Li Linhong,Wu Xiaolin,Su Ling,Raley Castle,Theobald Narda,Choi Uimook,Song Alexander H,Chan Andy,Pearl Jocelynn R,Paschon David E,Lee Janet,Newcombe Hannah,Koontz Sherry,Sweeney Colin,Shivak David A,Zarember Kol A,Peshwa Madhusudan V,Gregory Philip D,Urnov Fyodor D,Malech Harry L

Publisher

Springer Science and Business Media LLC

Subject

Biomedical Engineering,Molecular Medicine,Applied Microbiology and Biotechnology,Bioengineering,Biotechnology

Link

http://www.nature.com/articles/nbt.3513.pdf

Reference24 articles.

1. Biffi, A. et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341, 1233158 (2013).

2. Hacein-Bey-Abina, S. et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 363, 355–364 (2010).

3. Cavazzana-Calvo, M. et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288, 669–672 (2000).

4. Hacein-Bey-Abina, S. et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 348, 255–256 (2003).

5. Stein, S. et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat. Med. 16, 198–204 (2010).

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