Article
CFTR gene transfer with AAV improves early cystic fibrosis pig phenotypes.
JCI insight - 8 Sept 2016
Steines Benjamin, Dickey David D, Bergen Jamie, Excoffon Katherine Jda, 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
Abstract excerpt
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 capsids by replicating their genomes with helper adenovirus coinfection, we selected an adeno-associated virus (AAV)...
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