发明名称 |
INTRANASAL DELIVERY OF THERAPEUTIC ENZYMES TO THE CENTRAL NERVOUS SYSTEM FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES |
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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申请公布号 |
US2012288489(A1) |
申请公布日期 |
2012.11.15 |
申请号 |
US201213465575 |
申请日期 |
2012.05.07 |
申请人 |
HEALTH PARTNERS RESEARCH FOUNDATION;REGENTS OF THE UNIVERSITY OF MINNESOTA |
发明人 |
WOLF DANIEL A.;FREY, II WILLIAM H.;MCLVOR R. SCOTT;HANSON LEAH R. |
分类号 |
A61K38/43;A61K31/7088;A61K38/46;A61K38/47;A61P25/00;C12N9/16;C12N9/24;C12N15/864 |
主分类号 |
A61K38/43 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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