Systemic Gene Transfer to Skeletal Muscle Using Reengineered AAV Vectors

  • Jana L. Phillips
  • Julia Hegge
  • Jon A. Wolff
  • R. Jude Samulski
  • Aravind Asokan
Part of the Methods in Molecular Biology book series (MIMB, volume 709)


Gene therapy of musculoskeletal disorders warrants efficient gene transfer to a wide range of muscle groups. Reengineered adeno-associated viral (AAV) vectors that selectively transduce muscle tissue following systemic administration are attractive candidates for such applications. Here we provide examples of several lab-derived AAV vectors that display systemic tissue tropism in mice. Methods to evaluate the efficiency of gene transfer to skeletal muscle following intravenous or isolated limb infusion of AAV ­vectors in mice are discussed in detail.

Key words

AAV Reengineering Capsid Tropism Muscle Gene transfer Isolated limb infusion Gene therapy 


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Copyright information

© Springer Science+Business Media, LLC 2011

Authors and Affiliations

  • Jana L. Phillips
    • 1
  • Julia Hegge
    • 2
  • Jon A. Wolff
    • 2
  • R. Jude Samulski
    • 1
  • Aravind Asokan
    • 1
    • 3
  1. 1.Gene Therapy CenterUniversity of North Carolina at Chapel HillChapel HillUSA
  2. 2.Mirus BioCorporationMadisonUSA
  3. 3.Department of GeneticsUniversity of North Carolina at Chapel HillChapel HillUSA

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