Regents of the University of Minnesota;HealthPartners Research & Education
发明人:
Daniel A. Wolf,William H. Frey, II,R. Scott McIvor,Leah R. Hanson
申请号：
US14103597
公开号：
US09249424B2
申请日:
2013.12.11
申请国别(地区):
US
年份:
2016
代理人:
摘要：
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.
