An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque

Recombinant adeno-associated virus (rAAV)rh74.MCK.GALGT2 is a muscle-specific gene therapy that is being developed to treat forms of muscular dystrophy. Here we report on an isolated limb infusion technique in a non-human primate model, where hindlimb blood flow is transiently isolated using balloon...

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Publicado en:Mol Ther Methods Clin Dev
Main Authors: Xu, Rui, Jia, Ying, Zygmunt, Deborah A., Cramer, Megan L., Crowe, Kelly E., Shao, Guohong, Maki, Agatha E., Guggenheim, Haley N., Hood, Benjamin C., Griffin, Danielle A., Peterson, Ellyn, Bolon, Brad, Cheatham, John P., Cheatham, Sharon L., Flanigan, Kevin M., Rodino-Klapac, Louise R., Chicoine, Louis G., Martin, Paul T.
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2018
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6070685/
https://ncbi.nlm.nih.gov/pubmed/30073180
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.06.002
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