Intranasal delivery of AAV encoding therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
AssigneesRegents of the University of Minnesota, HealthPartners Research & Education
InventorsDaniel A. Wolf, William H. Frey, II, R. Scott McIvor, Leah R. Hanson
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Abstract
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.
Claims16
A method to prevent, inhibit or treat one or more neurological signs or symptoms associated with a deficiency in alpha-L-iduronidase, iduronate-2-sulfatase, N-acetyl-alpha-D-glucosaminidase, beta-galactosidase, or beta-glucuronidase in a mammal in, comprising:
- intranasally administering to the mammal a composition comprising an amount of a recombinant adeno-associated virus vector comprising an open reading frame encoding a lysosomal storage enzyme comprising alpha-L-iduronidase, iduronate-2-sulfatase, N-acetyl-alpha-D-glucosaminidase, beta-galactosidase, or beta-glucuronidase, effective to prevent, inhibit or treat the one or more neurological signs or symptoms associated with the deficiency.
Dependencies are inferred from the claim text.