FIELD: biotechnology.
SUBSTANCE: method of enhancing neurocognitive function in a mammal having a lysosomal storage disease is described, comprising: intrathecal administration to the mammal of a composition containing an amount of recombinant adeno-associated virus (rAAV) containing an open reading frame encoding alpha-L-iduronidase or iduronate-2-sulfatase effective to enhance neurocognitive function in a mammal compared to a mammal having a lysosomal storage disease that has not been administered rAAV, wherein the rAAV has capsid rAAV9 or rAAVrh10.
EFFECT: invention expands the arsenal of means for enhancing neurocognitive function in a mammal.
21 cl, 42 dwg, 8 ex
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Authors
Dates
2023-10-09—Published
2016-05-13—Filed