Gene therapy for amyotrophic lateral sclerosis and other...

A - Human Necessities – 61 – K

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Details

A61K 48/00 (2006.01) A61K 49/00 (2006.01) C12N 15/00 (2006.01) C12N 15/86 (2006.01) C12N 15/864 (2006.01) C12Q 1/68 (2006.01)

Patent

CA 2654292

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.

La présente invention concerne des procédés et des compositions permettant de traiter des troubles ou des lésions qui affectent la fonction et la régulation motrices chez un sujet. Selon un aspect de l'invention, un produit transgénique est libéré dans la moelle épinière d'un sujet par l'administration au cerveau d'un vecteur viral neurotrophique recombinant contenant le transgène. Le vecteur viral libère le transgène dans une région du cerveau qui est susceptible d'être infectée par le virus et qui exprime le produit génique viral recombinant encodé. L'invention concerne également des compositions permettant de libérer un produit transgénique dans la moelle épinière d'un sujet par l'administration au cerveau du sujet d'un vecteur viral neurotrophique recombinant contenant le transgène.

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