Article

DNA-PKcs Inhibition Improves Sequential Gene Insertion of the Full-Length <i>CFTR</i> cDNA in Airway Stem Cells

2024-08-12

Abstract excerpt

<h4>ABSTRACT</h4> Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene. Although many people with CF (pwCF) are treated using CFTR modulators, some are non-responsive due to their genotype or other uncharacterized reasons. Autologous airway stem cell therapies, in which the CFTR cDNA has been replaced, may enable a durable therapy for all pwCF. Pre...

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Literature Corpus work: 7d1641f4-9de9-57fb-93f2-05b5b50796ce
DOI: 10.1101/2024.08.12.607571

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