Gene therapy for neurometabolic disorders

Details Gene therapy for neurometabolic disorders Gene therapy for neurometabolic disorders

A - Human Necessities

61

K

A61K 48/00 (2006.01) C12N 15/63 (2006.01) C12N 15/74 (2006.01) C12N 15/86 (2006.01)

Patent

CA 2607173

The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.

L'invention porte sur des procédés et des compositions de traitement de troubles affectant le système nerveux central (CNS). Parmi ces troubles figurent des troubles neurométaboliques tels que les maladies lysosomales qui affectent le système nerveux central, par exemple la maladie de Niemann-Pick, ainsi que des troubles tels que la maladie d'Alzheimer. Les procédés de l'invention consistent à mettre en contact une terminaison axonale d'une neurone avec une composition contenant un virus associé aux adénovirus (AAV) transportant un transgène thérapeutique si bien que le vecteur AAV soit axonalement transporté de manière rétrograde et que le produit transgénique soit exprimé de manière distale au site d'administration.

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