Gene therapy for neurodegenerative disorders

Details Gene therapy for neurodegenerative disorders Gene therapy for neurodegenerative disorders

C - Chemistry, Metallurgy

12

N

C12N 15/864 (2006.01) A61K 48/00 (2006.01) A61P 21/00 (2006.01) C12N 7/01 (2006.01) C07K 14/47 (2006.01)

Patent

CA 2759801

Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed. Also disclosed is a self-complementary adeno-associated virus vector for treating motor neuron disorders such as spinal muscular atrophy, amytrophic lateral sclerosis, spinal bulbar muscular atrophy, spinal cerebellar ataxia, primary lateral sclerosis, and traumatic spinal cord injury.

Cette invention concerne des compositions et des méthodes de traitement des maladies touchant la fonction motrice et consécutives, par exemple, à une pathologie, à des lésions du cerveau et/ou de la moelle épinière. L'invention concerne également un vecteur de virus adéno-associé auto-complémentaire utilisé pour traiter les affections des motoneurones comme l'amyotrophie spinale, la sclérose latérale amyotrophique, l'amyotrophie bulbo-spinale, l'ataxie cérébelleuse, la sclérose latérale primitive et les lésions traumatiques de la moelle épinière.

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