Molecular Therapy
Volume 13, Issue 2, February 2006, Pages 241-249
Journal home page for Molecular Therapy

Review Article
Gene Therapy Strategies for Duchenne Muscular Dystrophy Utilizing Recombinant Adeno-associated Virus Vectors

https://doi.org/10.1016/j.ymthe.2005.11.001Get rights and content
Under a Creative Commons license
open archive

Abstract

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.

Keywords

dystrophin
adeno-associated virus
Duchenne muscular dystrophy
gene therapy
transsplicing
antisense RNA

Cited by (0)