FIELD: biotechnology.
SUBSTANCE: method for treating or preventing myotonic dystrophy type 1 (DM1) in an individual is described. Therapeutically effective amount of a cationic peptide-linked morpholino antisense oligonucleotide is systematically administered to an individual, said oligonucleotide comprising: (a) a sequence of a morpholino antisense oligonucleotide, complementary to at least 3 repeat sequences of poly-CUG in the 3'-untranslated region (UTR) of myotonic dystrophy protein kinase (DMPK) of the RNA transcript target; and (b) a spacer group linking the morpholino antisense oligonucleotide and the cationic peptide, the spacer group comprising
EFFECT: invention makes it possible to alleviate at least one symptom of DM1 in at least two muscles.
21 cl, 6 dwg, 4 ex
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