Treatment Of Lysosomal Storage Disorder Patients By Drug-enhanced Premature Stop Codon Read-through
Funder
National Health and Medical Research Council
Funding Amount
$431,764.00
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.
Development Of A Safe And Effective Treatment For Neuropathology In MPS IIIA.
Funder
National Health and Medical Research Council
Funding Amount
$665,320.00
Summary
MPS IIIA is an inherited disorder that results in progressive brain disease in affected children. The disorder cannot be treated at present because it has not been possible to find an effective way to deliver treatment to the brain. This project seeks to evaluate a method to overcome this problem. Findings in this project can be applied to other, similar disorders that affect the brain.