New Prime Editing Approach Corrects CFTR F508del Mutation in Human Lung Cells

Jul 10, 2024, 12:02 PM

Scientists have developed a prime editing approach that efficiently corrects the most common cystic fibrosis mutation in human lung cells, which could pave the way to a one-time treatment as effective as existing daily therapies. The systematic application of six recent prime editing developments has enabled the correction of the CFTR F508del mutation in primary airway cells from cystic fibrosis (CF) patients. This research, reported in Nature Biomedical Engineering, suggests the potential for a one-time treatment that addresses the root cause of the disease. While additional work lies ahead, these findings mark a significant step forward in the treatment of cystic fibrosis.