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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2016
Assuntos:
Research Article
Acesso em linha: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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