Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
AssigneeGenzyme Corporation
InventorsJames Dodge, Lamya Shihabuddin, Catherine O’Riordan
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AssigneeGenzyme Corporation
InventorsJames Dodge, Lamya Shihabuddin, Catherine O’Riordan
Estimating the term…
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.
1-9. (canceled)
A method to treat amyotrophic lateral sclerosis (ALS) in a subject, comprising administering a recombinant neurotrophic viral vector comprising a therapeutic transgene to at least one ventricle of the brain, whereby said transgene is expressed in a therapeutically effective amount;
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17-20. (canceled)
24-30. (canceled)
A method to treat amyotrophic lateral sclerosis in a human subject, comprising administering a recombinant AAV4 viral vector comprising a VEGF transgene to at least one ventricle of the brain selected from the group consisting of a lateral ventricle and the fourth ventricle, whereby said VEGF transgene is expressed in a therapeutically effective amount.
Dependencies are inferred from the claim text.