Duchenne muscular dystrophy (DMD) is a severe X-linked muscle-wasting disorder caused by mutations in the dystrophin gene that leads to progressive loss of mobility, cardiomyopathy and premature death. Despite recent advances in gene and exon-skipping therapies, treatment options remain limited and do not fully restore dystrophin function. Now, writing in Cell, Guo et al. describe an RNA-editing-based therapeutic strategy that harnesses endogenous RNA-editing enzymes to induce exon skipping and restore dystrophin expression. The approach produced durable therapeutic effects in non-human primate models and showed preliminary evidence of safety and efficacy in patients with DMD.Access optionsAccess Nature and 54 other Nature Portfolio journalsGet Nature+, our best-value online-access subscription27,99 € / 30 dayscancel any timeLearn moreSubscribe to this journalReceive 12 print issues and online access269,00 € per yearonly 22,42 € per issueLearn moreRent or buy this articlePrices vary by article typefrom$1.95to$39.95Learn morePrices may be subject to local taxes which are calculated during checkoutReferencesGuo, W. et al. Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans. Cell https://doi.org/10.1016/j.cell.2026.05.030 (2026)Download references