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Advances in Cell and Gene-based Therapies for Cystic Fibrosis Lung Disease

28 Feb 2012

Abstract excerpt

Cystic fibrosis (CF) is a disease characterized by airway infection, inflammation, remodeling, and obstruction that gradually destroy the lungs. Direct delivery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to airway epithelia may offer advantages, as the tissue is accessible for topical delivery of vectors. Yet, physical and host immune barriers in the lung present challenges for...

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PMID: 22371844
DOI: 10.1038/mt.2012.32

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