FIELD: biotechnology.
SUBSTANCE: disclosed is a recombinant genetic construct for overexpression of arylsulfatase A enzyme containing a promoter of a hybrid CMV enhancer/chicken β-actin and a codon-optimized sequence of the ARSA gene represented by SEQ ID NO: 1. Also disclosed is adeno-associated virus of serotype Olig001 for therapy of metachromatic leukodystrophy, containing recombinant genetic construct according to cl. 1.
EFFECT: invention enables therapy of metachromatic leukodystrophy.
2 cl, 5 dwg, 3 ex
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