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...去小宇宙查看完整单集简介
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