Investigation of Double-Stranded DNA Donors and CRISPR-Cas9 RNP for Universal Correction of Mutations Causing Cystic Fibrosis in Human Airway Cells
Vrishti Sinha, Paul George Ayoub, Colin J. Juett, Lindsay E. Lathrop, Ruth A. Foley, Ruby A. Sims, Joseph D. Long, Emily C. Duggan, Neil Fernandes, Beate Illek, Brigitte N. Gomperts, Steven John Jonas, Donald B. Kohn
This study demonstrates that low levels of CFTR integration can restore chloride ion currents in human airway cells using optimized nonviral CRISPR-Cas9 RNP and double-stranded DNA donors.
bioRxiv · Sep 21, 2025 · PreprintGenetics