Journal
MOLECULAR THERAPY
Volume 13, Issue 2, Pages 241-249Publisher
CELL PRESS
DOI: 10.1016/j.ymthe.2005.11.001
Keywords
dystrophin; adeno-associated virus; Duchenne muscular dystrophy; gene therapy; transsplicing; antisense RNA
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Funding
- NICHD NIH HHS [U54 HD47175] Funding Source: Medline
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Gene transfer vectors based on adeno-associated virus (AAV) are now widely used in the field of gene therapy. These vectors have been studied for their potential use in treating many diseases, among them the muscular dystrophies, the most common of which is Duchenne muscular dystrophy (DMD). Several recent advances in the areas of AAV serotype analysis, transgene engineering, and vector delivery to muscle, together with novel means of rescuing mutant mRNA transcripts, have yielded impressive results in animal models of DMD. This minireview focuses on these recent advances and their implications for potential treatments for DMD and other neuromuscular disorders.
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