SINGLE-CHEMICAL NUCLEIC ACID MOLECULES FOR GENE EXPRESSION CONTROL Russian patent published in 2017 - IPC C12N15/09 C12N15/113 A61K48/00 

FIELD: biotechnology.

SUBSTANCE: invention relates to biochemistry, in particular to a single-stranded nucleic acid molecule that inhibits target gene expression. The said molecule in the order from the 5'-end to the 3'-end contains the 5'-end portion Xc, the linker portion Lx, the inner portion Z, the linker portion Ly and the 3'-end portion Yc. At that, the inner region Z consists of the inner 5'-end portion X that is complementary to the 5'-end portion Xc and the inner 3'-end portion Y that is complementary to the 3'-end portion Yc. At that, Z consists of 19-30 bases and contains a sequence that suppresses the target gene expression. Z-(Xc+Yc) from 0 to 4 bases, Xc from 1 to 11 bases and Yc from 1 to 11 bases. This invention also discloses a method, application and composition for target gene expression inhibition, method, use and pharmaceutical composition for treatment of a disease caused by an increase in target gene expression using the above-mentioned nucleic acid molecule and the use of such molecule to induce RNA interference.

EFFECT: invention allows application of a single-stranded nucleic acid molecule for patients treatment, since it does not cause a side effect in the form of interferon induction.

32 cl, 32 dwg, 7 tbl, 25 ex