Therapy for CNS degeneration in MPS disorders that targets both glycosaminoglycan and ganglioside storage.

Funding Activity

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Funded Activity Summary

Children with seven of the eleven types of mucopolysaccharidosis (MPS) disorders exhibit a profound, irreversible neurological deterioration that manifests in infancy. This results from the continual buildup of undegraded sugar and fat in brain cells. The goal of this proposal is to prevent the accumulation of lipid alone or both lipid and sugar in the brain in order to alter the progression of neurological disease. Treatment will be assessed in mouse models of MPS.

Funded Activity Details

Start Date: 01-01-2009

End Date: 01-01-2011

Funding Scheme: NHMRC Project Grants

Funding Amount: $368,043.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Paediatrics

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

CNS disease | gangliosides | genetic disease | glycosaminoglycan biochemistry | learning and memory | lysosomal storage disorders | mucopolysaccharidosis | neurological degeneration | paediatric disease | therapy