title: Whole body skeletal muscle transduction in neonatal dogs with AAV-9.
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.
authors.affiliation: Department of Molecular Microbiology and Immunology, School of Medicine, The University of Missouri, One Hospital Drive, Columbia, USA.
authors.forename: Yongping
authors.initials: Y
authors.lastname: Yue
authors.forename: Jin-Hong
authors.initials: JH
authors.lastname: Shin
authors.forename: Dongsheng
authors.initials: D
authors.lastname: Duan
mesh_terms: D000469:Alkaline Phosphatase / Q000235:genetics*; D000818:Animals; D000831:Animals, Newborn; D000229:Dependovirus / Q000235:genetics*; D004285:Dogs; D017930:Genes, Reporter; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D018482:Muscle, Skeletal; D009137:Muscular Dystrophy, Animal / Q000235:genetics / Q000628:therapy*; D014161:Transduction, Genetic
chemical_list: D000469:Alkaline Phosphatase