FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology, specifically to RNA interference molecules (RNAi) targeted against the nucleotide sequence in Huntington disease, and methods of using said RNAi molecules for treating Huntington's disease. Described is a nucleic acid for use in treating a subject with Huntington's disease, said nucleic acid encoding an artificial primary miRNA transcript (pri-miRNA), consisting of, in order of location, 5'-flanking region, a non-guiding region, a loop region, a guide region and 3'-flanking region, wherein the guide area consists of SEQ ID NO: 37 (miHDss3), SEQ ID NO: 6 (miHDS1v5U) or SEQ ID NO: 7 (miHDS1v6A) and non-guiding region is at least 80 % complementary to the guide region.
EFFECT: invention can be used in treating Huntington's disease.
74 cl, 6 dwg, 1 ex
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