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...

詳細記述

保存先:
書誌詳細
出版年:JCI Insight
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: American Society for Clinical Investigation 2016
主題:
Research Article
オンライン･アクセス: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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