Studymoderate evidence
Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans.
Cell·July 9, 2026·PMID 42269605
Why it mattersThis represents a novel RNA editing approach that leverages endogenous ADAR enzymes, potentially offering a durable, single-dose therapy for DMD. The demonstration of functional improvements in both animal models and patients, without anti-dystrophin immune responses, addresses key limitations of current exon-skipping strategies.
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