Abstract
Gene therapy of muscular dystrophy requires systemic gene delivery to all muscles in the body. Adeno-associated viral (AAV) vectors have been shown to lead to body-wide muscle transduction after a single intravascular injection. Proof-of-principle has been demonstrated in mouse models of Duchenne muscular dystrophy and limb girdle muscular dystrophy. Before initiating clinical trials, it is important to validate these promising results in large animal models. More than a dozen canine muscular dystrophy models have been developed. Here, we outline a protocol for performing systemic AAV gene transfer in neonatal dogs. Implementing this technique in dystrophic dogs will accelerate translational muscular dystrophy research.
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Acknowledgments
The protocols were developed with the grant support from the National Institutes of Health (HL-91883, AR-49419 and AR-57209 to DD), the Muscular Dystrophy Association (DD) and the Parent Project Muscular Dystrophy (DD). We thank Drs. Dietrich Volkmann, Bruce Smith and Joe Kornegay for helpful discussion. We thank Robert J. McDonald, Jr., M.D. for the generous support to Duchenne muscular dystrophy research in the Duan lab.
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Yue, Y., Shin, JH., Duan, D. (2011). Whole Body Skeletal Muscle Transduction in Neonatal Dogs with AAV-9. In: Duan, D. (eds) Muscle Gene Therapy. Methods in Molecular Biology, vol 709. Humana Press. https://doi.org/10.1007/978-1-61737-982-6_21
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DOI: https://doi.org/10.1007/978-1-61737-982-6_21
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