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DNA-PKcs inhibition improves sequential gene insertion of the full-length CFTR cDNA in airway stem cells

17 Sept 2024

Abstract excerpt

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. Previously, CRISPR-Cas9...

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PMID: 39398224
DOI: 10.1016/j.omtn.2024.102339

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