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Base editing and nanoparticle transfection of airway cell types essential for treatment of cystic fibrosis

GSE324177 Homo sapiens Expression profiling by high throughput sequencing 6 samples Submitted 2026/03/11 Platform GPL24676
Summary
Cystic Fibrosis (CF) is a life-limiting genetic disorder caused by deleterious variants in the CFTR gene that results in altered mucous impairing the airway epithelia. Durable correction of these variants in airway cells remain a therapeutic challenge for ~10% of individuals unresponsive to CFTR modulators. A common disease-causing CFTR splice site variant 3120+1G>A was corrected in primary CF airway cells using base editor RNAs. Single-cell RNA sequencing revealed a remarkable increase in detectable CFTR transcript in most CF airway epithelial cell types resulting in notable enrichment of CFTR-expressing ionocytes and secretory goblet cells. Progenitor basal cell subtypes were edited but they decreased as a fraction of total cells and CFTR expressing cells compared to unedited cells.
Published in
Base editing and nanoparticle transfection of airway cell types essential for treatment of cystic fibrosis
Kavanagh EW, Joynt AT, Pion AR et al. · JCI insight 2026 · PMID 42100873 · doi:10.1172/jci.insight.198563
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Also filed as BioProject PRJNA1433566 and SRA study SRP681861. Searching any of these in the dataset finder brings you back here.

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