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Myogenesis pp 267-284 | Cite as

Recombinant Adeno-Associated Viral Vector Production and Purification

  • Jin-Hong Shin
  • Yongping Yue
  • Dongsheng Duan
Protocol
Part of the Methods in Molecular Biology book series (MIMB, volume 798)

Abstract

Gene delivery vectors based on recombinant adeno-associated virus (AAV) are powerful tools for studying myogenesis in normal and diseased conditions. Strategies have been developed to use AAV to increase, down-regulate, or modify expression of a particular muscle gene in a specific muscle, muscle group(s), or all muscles in the body. AAV-based muscle gene therapy has been shown to cure several inherited muscle diseases in animal models. Early clinical trials have also yielded promising results. In general, AAV vectors lead to robust, long-term in vivo transduction in rodents, dogs, and non-human primates. To meet specific research needs, investigators have developed numerous AAV variants by engineering viral capsid and/or genome. Here we outline a generic AAV production and purification protocol. Techniques described here are applicable to any AAV variant.

Key words

AAV Adeno-associated virus Muscle Gene therapy Gene transfer/delivery Serotype Muscular dystrophy Dystrophin Alkaline phosphatase 

Notes

Acknowledgments

The protocols were developed with the grant support from the National Institutes of Health (AR-49419 and HL-91883 to DD), the Muscular Dystrophy Association (DD), and the Parent Project for Muscular Dystrophy. We thank Duan lab members for helpful discussion.

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Copyright information

© Springer Science+Business Media, LLC 2012

Authors and Affiliations

  1. 1.Department of Molecular Microbiology and Immunology, School of MedicineUniversity of MissouriColumbiaUSA

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