Patent ID: 8637483
Filing Date: 2014-01-28
Classification: A61P,C12N

Abstract:
1. A combination of two or more antisense molecules selected from the following combinations: (a) an antisense oligonucleotide comprising SEQ ID NO: 31 and an antisense oligonucleotide comprising SEQ ID NO: 32; (b) an antisense oligonucleotide comprising SEQ ID NO: 33 and an antisense oligonucleotide comprising SEQ ID NO: 34; (c) an antisense oligonucleotide comprising SEQ ID NO: 35 and an antisense oligonucleotide comprising SEQ ID NO: 36; (d) an antisense oligonucleotide comprising SEQ ID NO: 39, an antisense oligonucleotide comprising SEQ ID NO: 40, and an antisense oligonucleotide comprising SEQ ID NO: 41; (e) an antisense oligonucleotide comprising SEQ ID NO: 42 and an antisense oligonucleotide comprising SEQ ID NO: 43; (f) an antisense oligonucleotide comprising SEQ ID NO: 44 and an antisense oligonucleotide comprising SEQ ID NO: 45; (g) an antisense oligonucleotide comprising SEQ ID NO: 46 and an antisense oligonucleotide comprising SEQ ID NO: 47; (h) an antisense oligonucleotide comprising SEQ ID NO: 48 and an antisense oligonucleotide comprising SEQ ID NO: 49; and (i) an antisense oligonucleotide comprising SEQ ID NO: 50 and an antisense oligonucleotide comprising SEQ ID NO: 51, wherein each of the antisense oligonucleotides comprises a modification to minimize or prevent cleavage by RNase H, and wherein the combination of antisense molecules is capable of binding to selected targets in dystrophin pre-mRNA to induce exon skipping in the human dystrophin gene.