GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS
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)
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.