Lung tissue-optimized gene editing in human cystic fibrosis models following topical application of lipid nanoparticles

Belal Tafech

doi:10.1016/j.jconrel.2025.114053

Lung tissue-optimized gene editing in human cystic fibrosis models following topical application of lipid nanoparticles

2025-07-18

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DOI：10.1016/j.jconrel.2025.114053

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• Developed and optimized a lipid nanoparticle (LNP)-based CRISPR delivery system for lung gene editing, achieving ~50 % editing efficacy in 2D models. • Editing efficacy dropped in complex human 3D cystic fibrosis tissue models but improved to ~12.7 % with dornase alpha pretreatment, showing clinical potential. • Achieved ~12 % gene correction in cystic fibrosis patient-derived cells with the CFTRR1162X point mutation, indicating promise for treating this specific untreatable cystic fibrosis variant.

Keywords:

Gene therapy

Gene editing

Lipid nanoparticles

Cystic fibrosis

Transmucosal delivery

Pulmonary gene delivery

CFTR

Lung tissue-optimized gene editing in human cystic fibrosis models following topical application of lipid nanoparticles

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