FIELD: biochemistry.
SUBSTANCE: invention relates to biochemistry. Antisense oligomer is described, which causes missing of 55th exon in the human dystrophin gene, consisting of a nucleotide sequence, complementary to any of the nucleotide sequences consisting of the 14–34th or 15–34th nucleotide, counting from 5'-the end of the 55th exon of the human dystrophin gene. Also disclosed is a pharmaceutical composition for the treatment of muscular dystrophy comprising, as an active ingredient, an antisense oligomer, or a pharmaceutically acceptable salt or hydrate thereof. In addition, there is provided a method of treating muscular dystrophy, comprising administering a therapeutically effective amount of a claimed oligomer.
EFFECT: invention expands the range of means for treatment of muscular dystrophy.
11 cl, 31 dwg, 15 tbl, 39 ex
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