FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology. A drug for the treatment of metachromatic leukodystrophy is described, including a recombinant adeno-associated serotype 9 virus containing a codon-optimized sequence of the ARSA gene represented by SEQ ID NO: 1, a drug for the treatment of metachromatic leukodystrophy consisting of mesenchymal stem cells genetically modified with a recombinant adeno-associated serotype 9 virus containing a codon-optimized sequence of the ARSA gene represented in SEQ ID NO: 1, and a method for treating metachromatic leukodystrophy. In one embodiment, the method for treatment of metachromatic leukodystrophy includes a single intravenous injection of the above drugs or a single intrathecal injection of a drug including a recombinant adeno-associated virus of serotype 9.
EFFECT: invention expands the arsenal of tools for gene and gene-cell therapy of metachromatic leukodystrophy.
3 cl, 6 dwg, 1 tbl, 3 ex
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