METHODS AND COMPOSITIONS FOR TREATING HUNTINGTON'S DISEASE Russian patent published in 2019 - IPC A61K48/00 A61K38/16 A61K38/54 C07H21/04 A61P25/14 

FIELD: medicine.

SUBSTANCE: invention refers to neurology, and can be used to reduce the expression of the mutant hauntingtin gene (mHTT) to reduce or prevent the formation of Htt aggregates in a subject with Huntington's disease (HD). That is ensured by administering a polynucleotide encoding a repressor of a mutant allele Htt (mHtt) into a striatum of a subject, wherein the repressor contains a "zinc finger" protein designated 30640, 30645 or 33074, wherein the polynucleotide comprises an adeno-associated viral vector (AAV) introduced in 2x10¹⁰ vector genomes, where the transcription factor repressor reduces mHtt expression by at least 85 % in HD-neurons in the subject's striatum.

EFFECT: invention provides reduction and/or elimination of aggregation of Huntingtin (Htt), reduced motor defects, increased cell activity (for example, ATP activity) and / or reduced apoptosis in a subject with HD.

7 cl, 7 dwg, 3 tbl, 6 ex