MODULATION OF HUNTINGTIN EXPRESSION Russian patent published in 2015 - IPC C12N15/11 C12N15/113 A61P25/14 

FIELD: chemistry.

SUBSTANCE: invention relates to biochemistry, particularly to compounds and compositions for reducing huntingtin expression. Disclosed are versions of a single-chain modified oligonucleotide which inhibits huntingtin expression. The oligonucleotide contains a gap segment consisting of ten deoxynucleosides, a 5'-flanking segment consisting of five nucleosides and a 3'-flanking segment consisting of five linked nucleosides. The gap segment is located between the 5'- and 3'-flanking segments, where all nucleosides of the flanking segments contain 2'-O-methoxyethyl-modified sugar. The internucleoside bonds in the gap segment, bonds which link the gap segment with the 5'- or 3'-flanking segment, and bonds for the outermost from the 5'-end and the outermost from the 3'-end nucleosides of each of the flanking segments are phosphorothioate bonds; the internucleoside bonds linking residual nucleosides of both flanking segments are phosphodiester bonds. All cytosines are 5-methylcytosines. The invention also discloses a composition and methods of treating, preventing, slowing down or alleviating Huntington's disease or symptoms thereof.

EFFECT: invention increases inhibiting activity on huntingtin expression.

10 dwg, 95 tbl, 21 ex