Prime editing efficiently corrects cystic fibrosis mutation in human lung cells
The approach targets the most common genetic cause of the disease and could enable a one-time treatment as effective as existing daily therapies.

Credit: Susanna Hamilton, Broad Communications
Funding
This work was supported in part by the National Institutes of Health and the Howard Hughes Medical Institute.
Paper cited
Sousa AA and Hemez C et al. Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells. Nature Biomedical Engineering. Online July 10, 2024. DOI:10.1038/s41551-024-01233-3.