Therapy for CNS degeneration in MPS disorders that targets both glycosaminoglycan and ganglioside storage. [ 2009 - 2011 ]

Also known as: Development of an effective therapy for brain disease in MPS children.

Research Grant

[Cite as]

Researchers: Dr Sharon Byers (Principal investigator) ,  Dr Janice Fletcher

Brief description 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.

Funding Amount $AUD 368,043.24

Funding Scheme NHMRC Project Grants

Notes Standard Project Grant

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