Now online! Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans
Circular ADAR-recruiting RNAs enable programmable exon skipping to bypass out-of-frame mutations in Duchenne muscular dystrophy and restore dystrophin expression. LEAPER 2.0 shows sustained efficacy in nonhuman primates and early clinical promise in patients, supporting circ-arRNA-mediated RNA editing as a translatable therapeutic strategy.
