[Cite as http://purl.org/au-research/grants/nhmrc/511321]
A/Pr Maria Fuller
Prof Douglas Brooks
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
Standard Project Grant