Patent ID: 9512424
Filing Date: 2016-12-06
CPC Classification: A61P,C12N

Claim Text:
1. An antisense oligomer which causes skipping of the 50th exon in a human dystrophin gene, consisting of a nucleotide sequence complementary to any one of the nucleotide sequences consisting of the 106th to the 126th, the 107th to the 127th, the 108th to the 127th, the 108th to the 128th, or the 109th to the 129th nucleotides, from the 5′ end of the human dystrophin gene's 50th exon, wherein the 50exon of the human dystrophin gene consists of position 1 to 109 of the nucleotide sequence SEQ ID NO: 3, and wherein the antisense oligomer is a morpholino opligomer or an oligonucleotide in which the sugar moiety and/or the phosphate-binding region of at least one nucleotide constituting the oligonucleotide is modified.