1200-circ-arRNA Guided Exon Skipping for DMD Therapy

Paper Talk · Jun 28, 2026 · 11m

This article introduces LEAPER 2.0, an innovative RNA editing platform designed to treat Duchenne muscular dystrophy (DMD). The technology utilizes circular ADAR-recruiting RNAs (circ-arRNAs) to induce programmable exon skipping, which restores the reading frame of the dystrophin gene. Research results demonstrate that this method effectively restores dystrophin protein expression in both nonhuman primates and patient-derived cells through a combination of chemical editing and physical interference. Long-term animal studies showed sustained improvements in muscle pathology and motor function…