FIELD: biochemistry.
SUBSTANCE: present invention relates to single-stranded modified oligonucleotides, capable of inhibiting expression of transthyretin in a cell. Said oligonucleotides have length from 15 to 30 linked nucleosides and can have a modified phosphorothioate bond, modified sugar and modified nucleobase, for example, 5-methylcytosine. Oligonucleotide according to present invention contains 5'-side segment linked to gap segment, which is linked with 3'-side segment. Present invention discloses a composition containing said oligonucleotide. Due to ability of said composition to inhibit expression of transthyretin, it is used for treating transthyretin amyloidosis, family amyloid polyneuropathy, family amyloid cardiomyopathy and senile systemic amyloidosis in humans.
EFFECT: present invention widens range of agents for inhibiting expression of transthyretin in a cell and treating transthyretin amyloidosis in humans.
21 cl, 95 tbl, 24 ex
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