Selection and evaluation of clinically relevant AAV variants in a xenograft liver model-Reference-Cited by-同舟云学术

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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Published:2013-12-25 Issue:7488 Volume:506 Page:382-386
ISSN:0028-0836
Container-title:Nature
language:en
Short-container-title:Nature

Author:

Lisowski Leszek,Dane Allison P.,Chu Kirk,Zhang Yue,Cunningham Sharon C.,Wilson Elizabeth M.,Nygaard Sean,Grompe Markus,Alexander Ian E.,Kay Mark A.

Publisher

Springer Science and Business Media LLC

Subject

Multidisciplinary

Link

http://www.nature.com/articles/nature12875.pdf

Reference35 articles.

1. Bainbridge, J. W. et al. Effect of gene therapy on visual function in Leber’s congenital amaurosis. N. Engl. J. Med. 358, 2231–2239 (2008)

2. Gaudet, D. et al. Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial. Gene Ther. 20, 361–369 (2013)

3. Bennett, J. et al. AAV2 gene therapy readministration in three adults with congenital blindness. Sci. Trans. Med. 4, 120ra115 (2012)

4. Nietupski, J. B. et al. Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression. Mol. Ther. 19, 1999–2011 (2011)

5. Jiang, H. et al. Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 108, 3321–3328 (2006)

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