Gene therapy for spinal cord disorders
AssigneeGenzyme Corporation
InventorsJames Dodge, Lamya Shihabuddin, Marco Passini, Seng H. Cheng, Catherine O'Riordan
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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 provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.
Claims10
A method to treat amyotrophic lateral sclerosis (ALS) in a subject, comprising administering a recombinant neurotropic viral vector comprising a therapeutic transgene to at least one region of the deep cerebellar nuclei region of the brain under conditions that favor expression of the transgene in a motor neuron distal to the site of administration, wherein the transgene product is delivered in a therapeutically effective amount to at least one subdivision of the spinal cord and/or at least one division of the brainstem, wherein said neurotropic viral vector is an adeno-associated viral vector comprising an AAV1 serotype capsid, and wherein the therapeutic transgene is insulin growth factor-1 (IGF-1).
Dependencies are inferred from the claim text.