New Lipid Nanoparticles and Prime Editing Correct CFTR F508del for Cystic Fibrosis

Jul 10, 2024, 12:02 PM

Scientists have developed new lipid nanoparticle designs that enable targeted in vivo lung editing in mice, potentially leading to new treatment options for cystic fibrosis. Recent advancements in prime editing, including the systematic application of six recent prime editing developments, have allowed for the efficient and functional correction of the CFTR F508del mutation, the predominant cause of cystic fibrosis, in primary airway cells from CF patients. This breakthrough, reported in Nature Biomedical Engineering, suggests the potential for a one-time prime editing treatment for cystic fibrosis that could be as effective as existing daily therapies.