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