Article
Widespread airway distribution and short-term phenotypic correction of cystic fibrosis pigs following aerosol delivery of piggyBac/adenovirus.
Nucleic acids research - 12 Oct 2018
Cooney Ashley L, Singh Brajesh K, Loza Laura Marquez, Thornell Ian M, Hippee Camilla E, Powers Linda S, Ostedgaard Lynda S, Meyerholz David K, Wohlford-Lenane Chris, Stoltz David A, B McCray Paul, Sinn Patrick L
Abstract excerpt
Cystic fibrosis (CF) is a common genetic disease caused by mutations in the gene coding for cystic fibrosis transmembrane conductance regulator (CFTR). Although CF affects multiple organ systems, chronic bacterial infections and inflammation in the lung are the leading causes of morbidity and mortality in people with CF. Complementation with a functional CFTR gene repairs this defect, regardless of the...
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