Patent ID: 8741863

Claim:
An antisense compound, comprising: an antisense oligonucleotide having 8-30 bases, with at least 8 contiguous bases being complementary to the polyCUG or polyCCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA in DM1 or DM2, respectively, and conjugated to the oligonucleotide, a cell-penetrating peptide having the sequence (RXRRBR) 2 XB, where R is arginine; B is β-alanine; and each X is independently —C(O)—(CH 2 ) n —NH—, where n is 4-6 (SEQ ID NO: 67), where the compound is effective to selectively block the sequestration of at least one of muscleblind-like 1 protein (MBNL1) and CUG-binding protein CUGBP in heart and quadricep muscle in a myotonic dystrophy animal model.