Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies

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 cap...

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Hlavní autoři: Wang, Zejing, Storb, Rainer, Halbert, Christine L, Banks, Glen B., Butts, Tiffany M., Finn, Eric E., Allen, James M., Miller, A. Dusty, Chamberlain, Jeffrey S., Tapscott, Stephen J.
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2012
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3412492/
https://ncbi.nlm.nih.gov/pubmed/22692496
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.111
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