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

Research Grant

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

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