ANTISENSE NUCLEIC ACID Russian patent published in 2020 - IPC C12N15/113 C07H21/02 C07H21/04 A61K31/70 C12N5/00 

FIELD: biotechnology.

SUBSTANCE: described is a group of inventions comprising an antisense oligomer with a base length of 14–32 to exclude exon 45 in the human dystrophin gene, a pharmaceutical composition for treating muscular dystrophy, method of treating muscular dystrophy by inducing exclusion of exon 45 in the human dystrophin gene, use of said antisense oligomer or its pharmaceutically acceptable salt or for preparing a pharmaceutical composition for treating muscular dystrophy and an antisense oligomer of 14–32 bases in length to exclude exon 45 in the human dystrophin gene for use in treating muscular dystrophy. In one embodiment, the antisense oligomer includes two coupled oligomeric links, where two oligomeric links are not adjacent to each other or do not overlap with each other.

EFFECT: invention extends the range of products for eliminating exon 45 in the human dystrophin gene.

21 cl, 14 tbl, 9 ex, 25 dwg