ANTISENSE NUCLEIC ACIDS Russian patent published in 2017 - IPC C12N15/113 A61P43/00 A61K31/7088 A61P21/04 A61K48/00 

FIELD: biotechnology.

SUBSTANCE: antisense oligomer is described which causes 50th exon skipping in the human dystrophin gene consisting of a nucleotide sequence complementary to any of the nucleotide sequences consisting of the 106 th - 126 th, 107 th - 127 th, 108 th - 127 th, 108 th - 128 th or 109 th - 129 th nucleotides, counting from the 5-terminus of the 50th' exon of human dystrophin gene. A pharmaceutical composition for muscular dystrophy treatment is also described, which comprises an antisense oligomer or a pharmaceutically acceptable salt or hydrate thereof, as an active ingredient.

EFFECT: invention expands the range of agents for muscular dystrophy treatment.

12 cl, 31 dwg, 15 tbl, 54 ex