Treatment of lysosomal storage disorder patients by drug-enhanced premature stop codon read-through

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/511321

Funding Status
Closed

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Funded Activity Summary

Lysosomal storage disorders are a devastating set of genetic diseases with very severe clinical symptoms. In this project, we will investigate a new treatment strategy that is non-invasive and that will be applicable for a wide range of lysosomal storage disorder patients. The therapy will over-ride the molecular genetic lesion and will be preferentially targeted for patients who are at the severe end of the clinical spectrum, where treatment options are currently limited.

Funded Activity Details

Start Date: 01-01-2008

End Date: 01-01-2010

Funding Scheme: NHMRC Project Grants

Funding Amount: $431,764.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Medical Genetics

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

Correction of molecular defect | Genetic disease | Lysosomal storage disease | Mental retardation | Novel therapeutic | Pre-clinical study | Treatment | lysosomal enzymes

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