MODULATING POLYNUCLEOTIDES Russian patent published in 2021 - IPC C12N15/113 C12N15/85 

FIELD: biotechnology.

SUBSTANCE: modulating polynucleotide is described for reducing or inhibiting the gene expression in a cell, where the specified modulating polynucleotide contains: (a) a stem and a loop that form a “stem-loop” structure, (b) the first flanking region located in the direction 5' from the specified accompanying chain, where the specified first flanking region contains 5'-end flanking sequence and 5'-end spacer sequence, where the first flanking region contains a nucleotide sequence, which is at least 90% identical to SEQ ID NO:5, and (c) the second flanking region located in the direction 3' from the specified guide chain, where the specified second flanking region contains 3'-end spacer sequence and 3'-end flanking region, where the specified second flanking region contains a nucleotide sequence, which is at least 90% identical to SEQ ID NO:21.

EFFECT: invention expands the arsenal of means for the treatment of central nervous system (CNS) disease.

28 cl, 2 dwg, 4 tbl, 4 ex