Researchers in Gene Therapy reported that adenine base editing can rescue a cryptic splice mutation in cardiac Fabry disease–relevant cells. The study details correction of an IVS4+919 G>A pathogenic change using ABE, aiming to restore proper transcript processing rather than replacing the gene. The work positions base editing as a mutation-specific strategy for Fabry, where standard approaches can be limited by delivery, lifelong dosing, or incomplete durability. Researchers emphasized cellular-level correction and downstream functional implications in the edited models. If the data translate, it could support a pathway toward targeted, one-time editing for defined Fabry mutation subsets rather than broader enzyme replacement strategies.