Intranasal delivery of AAV encoding therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases

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United States of America Patent

PATENT NO 9249424
APP PUB NO 20140171491A1
SERIAL NO

14103597

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Abstract

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The invention provides a method to prevent, inhibit or treat one or more neurological symptoms associated with a lysosomal storage disease in a mammal in need thereof, which includes intranasally administering to the mammal a composition comprising an effective amount of a lysosomal storage enzyme or a recombinant adeno-associated virus vector comprising an open reading frame encoding a lysosomal storage enzyme. Also provided are compositions and devices useful in the methods.

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Patent Owner(s)

  • HEALTHPARTNERS RESEARCH & EDUCATION;REGENTS OF THE UNIVERSITY OF MINNESOTA

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Frey,, II William H White Bear Lake, US 55 452
Hanson, Leah R Vadnais Heights, US 2 75
McIvor, R Scott St. Louis Park, US 36 295
Wolf, Daniel A Cambridge, US 2 75

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