VECTOR BASED ON ADENO-ASSOCIATED VIRUS FOR THERAPEUTIC DELIVERY TO CENTRAL NERVOUS SYSTEM Russian patent published in 2023 - IPC C12Q1/68 A61K38/47 

Abstract RU 2804953 C2

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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RU 2 804 953 C2

Authors

Makajvor, R. Skott

Belar, Lalita R.

Kozarski, Karen

Dates

2023-10-09Published

2016-05-13Filed