Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.

@article{Wang2012SuccessfulRD,
  title={Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.},
  author={Zejing Wang and Rainer F Storb and Christine L. Halbert and Glen B. Banks and Tiffany M Butts and Eric E. Finn and James M. Allen and A Dusty Miller and Jeffrey S. Chamberlain and Stephen J. Tapscott},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2012},
  volume={20 8},
  pages={1501-7}
}
Duchenne muscular dystrophy (DMD) is a fatal, X-linked muscle disease caused by mutations in the dystrophin gene. Adeno-associated viral (AAV) vector-mediated gene replacement strategies hold promise as a treatment. Studies in animal models and human trials suggested that immune responses to AAV capsid proteins and transgene products prevented efficient gene therapy. In this study, we used widespread intramuscular (i.m.) injection to deliver AAV6-canine micro-dystrophin (c-µdys) throughout a… CONTINUE READING
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A phase I dose-escalating study of AAV1–′-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C

S Herson, F Hentati, +3 authors F Leturcq
Mol Ther 19 (Suppl • 2011

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