Invention Grant
- Patent Title: Gene therapy for lysosomal storage diseases
- Patent Title (中): 溶酶体贮积病基因治疗
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Application No.: US12631336Application Date: 2009-12-04
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Publication No.: US08796236B2Publication Date: 2014-08-05
- Inventor: James Dodge , Seng Cheng
- Applicant: James Dodge , Seng Cheng
- Applicant Address: US MA Cambridge
- Assignee: Genzyme Corporation
- Current Assignee: Genzyme Corporation
- Current Assignee Address: US MA Cambridge
- Agency: Morrison & Foerster LLP
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A01N63/00

Abstract:
This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.
Public/Granted literature
- US20100173979A1 GENE THERAPY FOR LYSOSOMAL STORAGE DISEASES Public/Granted day:2010-07-08
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