CHROMOSOME 9 OPEN READING FRAME 72 GENE EXPRESSION MODULATORS AND APPLICATIONS THEREOF Russian patent published in 2024 - IPC A61K48/00 A61K38/00 A61K31/7088 A61P25/00 A61P25/08 C07K14/47 C12N15/62 C12N15/85 

FIELD: biotechnology.

SUBSTANCE: there are presented: a fusion protein for inhibiting transcription of a mutant allele of the C9orph72 gene in a patient in need thereof, containing a zinc finger protein (ZFP) DNA-binding domain and a transcription repressor domain, where the mutant allele C9orph72 contains more than 30 tandem repeats G₄C₂; nucleic acid construct for inhibiting transcription of the mutant allele of the C9orph72 gene in a patient in need thereof, comprising a coding sequence for a fused protein, and its use for the manufacture of a medicament for treating a patient; a host cell containing a nucleic acid construct; recombinant virus for inhibiting transcription of the mutant allele of the C9orph72 gene in a patient in need thereof, containing a nucleic acid construct. Also disclosed is a pharmaceutical composition for treating a patient suffering from a disorder selected from amyotrophic lateral sclerosis and familial frontotemporal dementia, comprising a nucleic acid construct; a method of inhibiting transcription of the mutant allele of the C9orph72 gene in a human cell and a method of treating a patient suffering from a disorder selected from amyotrophic lateral sclerosis and familial frontotemporal dementia.

EFFECT: invention is applicable for treating patients suffering a C9orph72-related disease, such as amyotrophic lateral sclerosis or frontotemporal dementia.

38 cl, 22 dwg, 1 tbl, 4 ex