FIELD: biotechnology.
SUBSTANCE: described is a group of inventions, including a splicing-switching oligonucleotide, a composition for treating or preventing Duchenne muscular dystrophy or Becker muscular dystrophy, containing a therapeutically effective amount of the above oligonucleotide, an application of the oligonucleotide or the composition for producing a medicinal product for treating or preventing Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy, and a method for preventing or treating delay of Duchenne muscular dystrophy or Becker muscular dystrophy, involving introduction of an effective amount of the oligonucleotide or the composition to the subject. In one implementation variant, the oligonucleotide has a length from 16 to 25 nucleotides and is complementary to at least part of the exons 44, 45, or 47 to 53 of the pre-mRNA of dystrophin.
EFFECT: invention expands the range of means for treating or preventing Duchenne muscular dystrophy or Becker muscular dystrophy.
19 cl, 9 dwg, 5 tbl, 4 ex
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