FIELD: gene therapy.
SUBSTANCE: disclosed is an expression cassette for treating Huntington's disease, comprising a promoter consisting of nucleic acid of 500 to 1700 nucleotides in length, which is at least 98 % identical to a polynucleotide sequence of at least 500 nucleotides in SEQ ID NO: 1, SEQ ID NO: 2 or SEQ ID NO: 7. Promoter is operably linked to a given segment of DNA containing a therapeutic gene which codes a protein or a heterologous RNA transcript. Also disclosed are an expression vector, a transformed mammalian cell comprising an expression cassette or an expression vector, method for production thereof.
EFFECT: use of an expression cassette for expressing a therapeutic gene in the onset or pathological progression of Huntington's disease activates and/or enhances expression of said therapeutic gene.
20 cl, 4 dwg, 1 ex
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