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INTRANASAL DELIVERY OF THERAPEUTIC ENZYMES TO THE CENTRAL NERVOUS SYSTEM FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES
专利权人:
R. Scott Mclvor;William H. Frey, II;Daniel A. Wolf;Leah R. Hanson
发明人:
Daniel A. Wolf,William H. Frey, II,R. Scott Mclvor,Leah R. Hanson
申请号:
US13465575
公开号:
US20120288489A1
申请日:
2012.05.07
申请国别(地区):
US
年份:
2012
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.
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