COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF DYSTROPHIC MIOTONIN-PROTEINKINASE (DMPK) Russian patent published in 2019 - IPC C12N15/113 A61K48/00 

FIELD: biotechnologies.

SUBSTANCE: invention relates to the biotechnology. Described are compounds for reducing DMPK mRNA expression containing a modified oligonucleotide consisting of 10–30 linked nucleosides and having at least one modified sugar component, a modified internucleoside bond and/or a modified nucleotide base, wherein said nucleotide sequence of the modified oligonucleotide is at least 90 % complementary to SEQ ID NO: 1 and/or SEQ ID NO: 2 throughout the length of the modified oligonucleotide. Also described is a compound for reducing DMPK mRNA expression containing a modified oligonucleotide having a nucleotide sequence, given in SEQ ID NO: 30, wherein the modified oligonucleotide consists of 16 bound nucleosides and contains: a gap segment consisting of eight bound deoxynucleosides; segment 5' wing, consisting of four bound nucleosides and having motif E-E-KK in 5' wing; segment 3' wing, consisting of four bound nucleosides and having motif KK-E-E in 3' wing; wherein gap segment is located between segment 5' wings and segment 3' wings; wherein each E is 2'-O-methoxyethyl sugar and each K is cet-sugar; wherein each internucleoside linkage is a thiophosphate internucleoside bond and wherein each cytosine residue is 5-methylcytosine. Presented a composition and a method for treating type 1 myotonic dystrophy in an animal.

EFFECT: invention enables to reduce expression of mRNA and DMPK protein in an animal.

28 cl, 20 ex, 32 tbl