FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology, specifically to gene therapy drugs based on β-hexosaminidase A, and can be used for treating the lysosomal Tay-Sachs accumulation disease (TSD). An expression cassette containing codon-optimized nucleotide sequences of HEXA and HEXB genes, separated by an IRES nucleotide sequence or coding P2A, E2A, F2A or T2A peptide, is designed for expression of β-hexosaminidase A in a mammalian cell. Expression cassette is used in a lentiviral expression vector in a pharmaceutical composition.
EFFECT: invention enables recovering the disturbed expression and/or function of the enzyme β-hexosaminidase A in the central nervous system, which has a positive effect in the patients suffering the Tay-Sachs disease.
2 cl, 5 dwg
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