A new Science Translational Medicine report described base-editing approaches that functionally correct the CFTR 1717-1G>A splicing mutation in patient-derived cell models. The target mutation is associated with severe cystic fibrosis and has largely lacked approved CFTR modulator options due to minimal protein production. Researchers from the University of Trento used an adenine base editing strategy leveraging the SpRY-ABE9 system, delivered with optimized mRNA and sgRNA constructs. The study reported functional correction in patient-derived airway epithelial cells with up to ~30% target DNA editing in cellular models and minimal off-target effects. The results expand base-editing feasibility for rare “untreatable” splicing genotypes and may broaden the addressable CF population as delivery platforms and editing systems continue to mature toward clinical translation.