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0This 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 without causing adverse immune reactions. Furthermore, first-in-human clinical data indicate that the treatment is safe and produces dose-dependent benefits in patients' respiratory and physical capabilities. Ultimately, the study presents a promising, durable therapeutic strategy that bypasses the limitations of traditional gene-editing and oligonucleotide therapies.
References:
Guo W, Tang H, Yi Z, et al. Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans[J]. Cell, 2026.
