ANTISENSE NUCLEIC ACIDS Russian patent published in 2015 - IPC C12N15/113 C07H21/04 A61K31/7125 

FIELD: medicine, pharmaceutics.

SUBSTANCE: group of inventions relates to the field of biotechnology, in particular to antisense oligomers, used as an active ingredient in pharmaceutical compositions for treating muscular dystrophy. An antisense oligomer consists of a nucleotide sequence, 100% complementary nucleotide target-sequence or nucleotide sequence in which 1 or 2 nucleotides, which are not complementary to the nucleotide target-sequence, are contained in the nucleotide sequence, 100% complementary to the nucleotide target-sequence. The nucleotide target-sequence represents any of the sequences, consisting of nucleotides from 32-nd to 56-th or from 36-th to 56-th from 5'-end of 53-rd exon in the human dystrophin gene. 53-rd exon in the human dystrophin gene possesses the nucleotide sequence, selected from (a) and (b): (a) nucleotide sequence SEQ ID NO: 1 and (b) nucleotide sequence, possessing at least 90% identity with the nucleotide sequence SEQ ID NO: 1.

EFFECT: said oligomer effectively induces skipping of 53-rd exon in the human dystrophin gene.

13 cl, 19 dwg, 7 tbl, 6 ex