grant

Treatment of lysosomal storage disorder patients by drug-enhanced premature stop codon read-through [ 2008 - 2010 ]

Also known as: A new therapy for lysosomal storage disorder patients

Research Grant

[Cite as http://purl.org/au-research/grants/nhmrc/511321]

Researchers: A/Pr Maria Fuller (Participant) Prof Douglas Brooks (Principal investigator)

Brief description 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.

Funding Amount $AUD 431,764.08

Funding Scheme NHMRC Project Grants

Notes Standard Project Grant

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