Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders

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.

We claim: 
     
         1 - 9 . (canceled) 
     
     
         10 . 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; wherein the transgene is VEGF and the viral vector is an adeno-associated (AAV) vector. 
     
     
         11 . (canceled) 
     
     
         12 . The method of  claim 10  wherein the viral vector is AAV4. 
     
     
         13 . (canceled) 
     
     
         14 . The method of  claim 10  wherein the viral vector is administered by direct injection into a ventricle of the brain. 
     
     
         15 . The method of  claim 10  wherein the viral vector is administered by direct injection into a lateral ventricle of the brain. 
     
     
         16 . The method of  claim 10  wherein the viral vector is administered by direct injection into the fourth ventricle of the brain. 
     
     
         17 - 20 . (canceled) 
     
     
         21 . The method of  claim 10  wherein said subject is a mammal. 
     
     
         22 . The method of  claim 21 , wherein said mammal is selected from the group consisting of a rodent, a murine, a simian, and a human. 
     
     
         23 . The method of  claim 10 , wherein said subject is a human patient. 
     
     
         24 - 30 . (canceled) 
     
     
         31 . The method of  claim 10 , wherein the viral vector is administered by direct injection into both a lateral ventricle of the brain and the fourth ventricle of the brain. 
     
     
         32 . 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.