METHODS AND COMPOSITIONS FOR TREATMENT OF HUNTINGTON'S CHOREA Russian patent published in 2022 - IPC C12N15/63 C07H21/02 

FIELD: biotechnology.

SUBSTANCE: polynucleotide is described for modulating the expression of a huntingtin (HTT) gene, containing a sequence of a semantic chain and a sequence of an anti-semantic chain of miRNA-duplex, where the sequence of the semantic chain contains at least 19 sequential nucleotides from a sequence of the semantic chain SEQ ID NO:168 or 171, and the sequence of the anti-semantic chain contains at least 19 sequential nucleotides from a sequence of the anti-semantic chain SEQ ID NO:7. Polynucleotide is also described for modulating the expression of a huntingtin (HTT) gene, containing a sequence of a semantic chain and a sequence of an anti-semantic chain of miRNA-duplex, where the sequence of the semantic chain contains at least 19 adjacent nucleotides from a sequence of the semantic chain SEQ ID NO:133, and the sequence of the anti-semantic chain contains at least 19 adjacent nucleotides from a sequence of the anti-semantic chain SEQ ID NO:9. A viral genome AAV is also described for modulating the expression of a huntingtin (HTT) gene, containing a nucleic acid sequence located between two inverted terminal repeats (ITR), encoding the specified polynucleotide. Polynucleotide for modulating the expression of HTT gene, containing any of sequences SEQ ID NO:349, 368, 347, is also presented. A corresponding particle of a recombinant adeno associated virus is also described. A pharmaceutical composition for the treatment of Huntington’s disease (HD), containing the specified polynucleotide, the viral genome AAV or the recombinant AAV particle and a pharmaceutically acceptable filler, is presented. A corresponding method for inhibiting the expression of HTT gene, mRNA and/or protein in a cell and a method for the treatment of Huntington’s disease (HD) in an individual are presented.

EFFECT: invention expands the arsenal of means for the treatment of Huntington’s disease.

70 cl, 27 tbl, 8 ex