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Article

Advances in gene therapy for cystic fibrosis lung disease.

Human molecular genetics - 1 Oct 2019

Yan Ziying, McCray Paul B, Engelhardt John F

Abstract excerpt

Cystic fibrosis (CF) is a multiorgan recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene therapy efforts have focused on treating the lung, since it manifests the most significant life-threatening disease. Over two decades have past since the first CF lung gene therapy trials and significant advances in the therapeutic implementation of...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 31332440
DOI: 10.1093/hmg/ddz139
PMCID: PMC6796993

Genes

CFTR

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