Article

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

2025-11-06

Abstract excerpt

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 was corrected in primary CF airway cells using base edit...

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Literature Corpus work: be71496d-3003-5af7-bed7-b524db608646
DOI: 10.1101/2025.11.06.687048

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