FIELD: biotechnology.
SUBSTANCE: described is a group of inventions comprising a compound for inducing exon 51 skipping in dystrophin pre-mRNA, a composition for inducing exon 51 skipping in dystrophin pre-mRNA, method of preventing, treating and/or slowing down Duchenne muscular dystrophy (DMD), involving administering a compound or composition to a subject and a method for skipping exon 51 in dystrophin pre-mRNA. In one embodiment, the compound comprises a first and a second antisense oligonucleotide (AON) connected to each other by a binding fragment, wherein said first antisense oligonucleotide (AON) is complementary to at least a portion of SEQ ID NO: 3, and wherein said second antisense oligonucleotide (AON) is complementary to at least a portion of SEQ ID NO: 4, wherein SEQ ID NO: 3 and 4 are located in exon 51 of dystrophin pre-mRNA.
EFFECT: invention extends the range of agents for inducing exon 51 skipping in dystrophin pre-mRNA.
19 cl, 2 dwg, 4 tbl, 2 ex
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