Gene therapy strategies for Duchenne muscular dystrophy utilizing recombinant adeno-associated virus vectors.

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.

• Publication year

  2006

• Venue

  Molecular Therapy

• Publication date

  2006-02-01

• Fields of study

  Medicine

• Identifiers
  DOI 10.1016/J.YMTHE.2005.11.001PMID 16361117
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

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