FIELD: medicine.
SUBSTANCE: invention relates to medicine and relates to a method for the treatment of Tay-Sachs disease and Sandhoff disease (TSD and SD) using genetically modified human mesenchymal stem cells with overexpression of β-hexosaminidase A, which consists in the fact that mesenchymal stem cells previously transduced with recombinant lenses are injected intravenously LV-HEXA and LV-HEXB or adeno-associated viruses AAV-HEXA and AAV-HEXB encoding genes for the enzyme β-hexosaminidase A.
EFFECT: invention ensures the restoration of the deficiency of the HexA enzyme and its enzymatic activity, thereby helping to slow down the progression of the TSD and SD.
1 cl, 6 dwg, 1 ex
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