Methods for the treatment of lysosomal storage disorders

Details Methods for the treatment of lysosomal storage disorders Methods for the treatment of lysosomal storage disorders

A - Human Necessities

61

K

A61K 35/30 (2006.01) A61P 25/28 (2006.01)

Patent

CA 2593110

Provided herein are methods for the treatment of lysosomal storage disorders characterized by a missing or defective secreted lysosomal enzyme. Such lysosomal storage disorders include, but are not limited to neuronal ceroid lipofuscinoses. The disclosed methods involve the transplantation of human multipotent neural stem cells into the CNS of patients suffering from the lysosomal storage disorder. Also provided herein are methods of reversing or slowing the progression of neurodegeneration in patients suffering from or at risk of developing neuronal ceroid lipofuscinoses.

L'invention concerne des procédés de traitement des troubles de stockage lysosomal caractérisés par une enzyme lysosomale sécrétée manquante ou défectueuse. Ces troubles englobent entre autres la céroïde-lipofuscinose neuronale. On décrit des procédés reposant sur la transplantation de cellules souches neuronales multipotentes humaines dans le système nerveux central de patients qui souffrent de ce type de trouble, et des procédés de réversion ou de ralentissement de la neurodégénérescence chez des patients souffrant ou risquant de souffrir d'une céroïde-lipofuscinose neuronale.

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