Article

Investigation of Double-Stranded DNA Donors and CRISPR-Cas9 RNP for Universal Correction of Mutations Causing Cystic Fibrosis in Human Airway Cells

2025-09-21

Abstract excerpt

<h4>ABSTRACT</h4> Cystic fibrosis (CF) is a devastating genetic disease caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator ( CFTR ) gene. As morbidity and mortality from CF results from a lack of mucus clearance that leads to chronic bacterial infections and progressive loss of lung function, site-specific insertion of a CFTR cDNA into the endogenous CFTR locus in airway basal stem...

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Literature Corpus work: a9b763f9-1126-5749-9a6f-0c0bae42f65e
DOI: 10.1101/2025.09.21.677613

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