Clinical trial registries and scientific publications

New Gene Editing Advances Show Promise for Treating CFTR F508del Mutation in Cystic Fibrosis

Jul 10, 2024, 12:02 PM

Recent advancements in gene editing have shown promising results in treating cystic fibrosis (CF). Scientists have developed a prime editing approach that efficiently corrects the most common CF mutation, CFTR F508del, in primary airway cells from CF patients. This method could pave the way for a one-time treatment as effective as existing daily therapies. Additionally, new lipid nanoparticle designs have enabled targeted in vivo lung editing in mice, potentially leading to new treatment options for CF. Researchers are optimistic about the potential of these developments to offer a permanent solution for CF patients.