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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Pubblicato in:JCI Insight
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society for Clinical Investigation 2016
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Research Article
Accesso online: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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