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Article

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

11 Jun 2019

Abstract excerpt

Abstract 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 surface. Adeno-associated virus (AAV) is a useful vector for gene therapy because of its low immunogenicity...

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PMID: 31184507
DOI: 10.1165/rcmb.2019-0006oc

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