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New #Cell paper: Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans. #Myoblue tinyurl.com/nvb5zftp
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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 i...
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Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans
Matthew Alexander, PhD