Invention Grant
- Patent Title: Intranasal delivery of AAV encoding therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
- Patent Title (中): 鼻内递送AAV编码治疗酶到中枢神经系统用于治疗溶酶体贮积病
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Application No.: US14103597Application Date: 2013-12-11
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Publication No.: US09249424B2Publication Date: 2016-02-02
- Inventor: Daniel A. Wolf , William H. Frey, II , R. Scott McIvor , Leah R. Hanson
- Applicant: Regents of the University of Minnesota , HealthPartners Research & Education
- Applicant Address: US MN Minneapolis US MN Bloomington
- Assignee: Regents of the University of Minnesota,HealthPartners Research & Education
- Current Assignee: Regents of the University of Minnesota,HealthPartners Research & Education
- Current Assignee Address: US MN Minneapolis US MN Bloomington
- Agency: Schwegman Lundberg & Woessner, P.A.
- Main IPC: C12N15/86
- IPC: C12N15/86 ; A61K38/46 ; A61K38/47 ; C12N9/24 ; C12N9/18 ; A61K9/00
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.
Public/Granted literature
Information query
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