MODULATION OF HUNTINGTIN EXPRESSION Russian patent published in 2021 - IPC C12N15/113 A61K31/7125 C07H21/04 A61P25/28 

FIELD: biotechnology; medicine.

SUBSTANCE: group of inventions relates to biotechnology and medicine. Compounds and compositions for attenuating the expression of mRNA and huntingtin protein in animals are proposed. The compound contains a modified oligonucleotide consisting of 18 consecutive nucleic bases, given in the sequence SEQ ID NO: 33, where the modified oligonucleotide has a gap segment located between the 5’-flanking segment and the 3’-flanking segment, each nucleoside in the 5’-and 3’ - flanking segments contains a modification of 2’ - MOE, the internucleoside bonds in the gapmer are phosphorothioate (P=S), and all the cytosines in the gapmer are 5-methylcytosines.

EFFECT: сompounds and pharmaceutical compositions based on it are applicable for treatment or prevention of Huntington's disease, and are characterized by high neurotransmission in vivo and a low IC₅₀ value.

7 cl, 10 dwg, 93 tbl, 21 ex