A Novel AAV-mediated Gene Delivery System Corrects CFTR Function in Pigs

Cystic fibrosis is an autosomal-recessive disease that is caused by a mutant CFTR (cystic fibrosis transmembrane conductance regulator) gene and is characterized by chronic bacterial lung infections and inflammation. Complementation with functional CFTR normalizes anion transport across the airway s...

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Bibliographic Details
Published in:Am J Respir Cell Mol Biol
Main Authors: Cooney, Ashley L., Thornell, Ian M., Singh, Brajesh K., Shah, Viral S., Stoltz, David A., McCray, Paul B., Zabner, Joseph, Sinn, Patrick L.
Format: Artigo
Language:Inglês
Published: American Thoracic Society 2019
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Original Research
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6890402/
https://ncbi.nlm.nih.gov/pubmed/31184507
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1165/rcmb.2019-0006OC
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