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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Yayımlandı:Mol Ther Methods Clin Dev
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: American Society of Gene & Cell Therapy 2018
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Article
Online Erişim: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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