Gene therapy for neurometabolic disorders
AssigneeGenzyme Corporation
InventorsMarco A. Passini, James Dodge
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Abstract
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.
Claims9
A method of treating Alzheimer's disease in a mammal comprising:
- administering a composition comprising an AAV vector having an AAV serotype 1 capsid encoding for a biologically active metalloendopeptidase molecule to the deep cerebellar nuclei of the cerebellum in the disease compromised central nervous system (CM) of the mammal with Alzheimer's disease, under conditions whereby said injected AAV vectors transduce cells located at a distal site in the central nervous system and said encoded biologically active metalloendopeptidase is expressed at a therapeutic level.
Dependencies are inferred from the claim text.