Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector-Reference-Cited by-同舟云学术

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Published:2012-02 Issue:2 Volume:20 Page:443-455
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Bowles Dawn E,McPhee Scott WJ,Li Chengwen,Gray Steven J,Samulski Jade J,Camp Angelique S,Li Juan,Wang Bing,Monahan Paul E,Rabinowitz Joseph E,Grieger Joshua C,Govindasamy Lakshmanan,Agbandje-McKenna Mavis,Xiao Xiao,Samulski R Jude

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

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4. rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice;Gregorevic;Nat Med,2006

5. Systemic human minidystrophin gene transfer improves functions and life span of dystrophin and dystrophin/utrophin-deficient mice;Wang;J Orthop Res,2009

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