Invention Grant
- Patent Title: Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
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Application No.: US16546070Application Date: 2019-08-20
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Publication No.: US11554161B2Publication Date: 2023-01-17
- Inventor: James Dodge , Lamya Shihabuddin , Catherine O'Riordan
- Applicant: Genzyme Corporation
- 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 ; C12N15/79 ; C07H21/04 ; A61K38/30 ; C12N15/864

Abstract:
This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord 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's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.
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