DMPK and Duchenne muscular dystrophy: DM1is caused by an abnormal number of CUG triplet repeats in the myotonicdystrophy protein kinase gene (DMPK), reducing muscleblind-like protein(MBNL) activity and disrupting muscle development.206 AOC 1001 is designed to reduce DMPK levels and CUG tripletrepeats so that MBNL can perform normally.205 Avidity Biosciences has also developed an AOC to treat DMD.205 The oligonucleotides are designed to promoteskipping of specific exons to produce dystrophin in patients withDMD.