Whole Body Skeletal Muscle Transduction in Neonatal Dogs with AAV-9
- 8 December 2010
- book chapter
- protocol
- Published by Springer Science and Business Media LLC in Methods in molecular biology (Clifton, N.J.)
- Vol. 709, 313-329
- https://doi.org/10.1007/978-1-61737-982-6_21
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.This publication has 25 references indexed in Scilit:
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