FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology. Isolated nucleic acid is proposed to reduce the expression of the pathogenic huntingtin protein. The isolated nucleic acid contains a transgene encoding one or more mature single-stranded microRNAs, in which the nucleotide sequence of the transgene encoding each mature single-stranded microRNA contains the sequence specified under SEQ ID NO: 7, and is flanked by a heterologous spanning sequence of microRNAs. A vector for reducing the expression of the pathogenic huntingtin protein, which contains isolated nucleic acid. Recombinant AAV (rAAV) to reduce the expression of the pathogenic huntingtin protein (NTT), where the specified rAAV contains (i) capsid protein and (ii) isolated nucleic acid.
EFFECT: invention reduces the expression of the pathogenic huntingtin protein in patients with Huntington's disease.
22 cl, 55 dwg, 6 tbl, 4 ex
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