grant

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.

Funded by National Health and Medical Research Council
Managed by The University of Adelaide
Managed by University of Adelaide
Provided by   National Health and Medical Research Council

Research Grant

[Cite as http://purl.org/au-research/grants/nhmrc/565080]

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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321019 | CNS disease | gangliosides | genetic disease | glycosaminoglycan biochemistry | learning and memory | lysosomal storage disorders | mucopolysaccharidosis | neurological degeneration | paediatric disease | therapy |
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