Article

Gene-Corrected Basal Cells Restore CFTR In Vitro; Transplants Regenerate Epithelium in a Preclinical Sinus Model

2025-07-21

Abstract excerpt

<h4>Background</h4> Cystic fibrosis (CF) is caused by mutations in the CFTR gene, leading to epithelial dysfunction and progressive lung disease. Although CFTR modulators have transformed care, ∼10% of people with CF remain without effective therapy. Durable, mutation-agnostic approaches are urgently needed. <h4>Method</h4> We used a lentiviral (LV) vector to deliver wild-type CFTR to airway basal cells deriv...

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Literature Corpus work: 7aa12e3c-8849-5668-817e-3663cc8f54f1
DOI: 10.1101/2025.07.21.666023

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