Whole body skeletal muscle transduction in neonatal dogs with AAV-9.
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ABSTRACT:
SUBMITTER: Yue Y
PROVIDER: S-EPMC3118043 | biostudies-literature | 2011
REPOSITORIES: biostudies-literature
Publications
Whole body skeletal muscle transduction in neonatal dogs with AAV-9.
Yue Yongping Y Shin Jin-Hong JH Duan Dongsheng D
Methods in molecular biology (Clifton, N.J.) 20110101
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 dy ...[more]
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