CFTR gene transfer with AAV improves early cystic fibrosis pig phenotypes

The physiological components that contribute to cystic fibrosis (CF) lung disease are steadily being elucidated. Gene therapy could potentially correct these defects. CFTR-null pigs provide a relevant model to test gene therapy vectors. Using an in vivo selection strategy that amplifies successful c...

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Udgivet i:JCI Insight
Main Authors: Steines, Benjamin, Dickey, David D., Bergen, Jamie, Excoffon, Katherine J.D.A., Weinstein, John R., Li, Xiaopeng, Yan, Ziying, Abou Alaiwa, Mahmoud H., Shah, Viral S., Bouzek, Drake C., Powers, Linda S., Gansemer, Nicholas D., Ostedgaard, Lynda S., Engelhardt, John F., Stoltz, David A., Welsh, Michael J., Sinn, Patrick L., Schaffer, David V., Zabner, Joseph
Format: Artigo
Sprog:Inglês
Udgivet: American Society for Clinical Investigation 2016
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5033908/
https://ncbi.nlm.nih.gov/pubmed/27699238
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.88728
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