Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler–Najjar Syndrome Type I-Reference-Cited by-同舟云学术

Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler–Najjar Syndrome Type I

Published:2017-08-14 Issue:10 Volume:24 Page:649-660
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Bočkor L,Bortolussi G,Iaconcig A,Chiaruttini G,Tiribelli C,Giacca M^ORCID,Benvenuti F,Zentilin L,Muro A F

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.nature.com/articles/gt201775.pdf

Reference73 articles.

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4. Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011; 365: 2357–2365.

5. Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014; 371: 1994–2004.

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