Targeting Pathogenic TAR DNA-binding Protein 43 To Treat Frontotemporal Dementia And Motor Neuron Disease
Funder
National Health and Medical Research Council
Funding Amount
$687,444.00
Summary
Frontotemporal dementia and motor neuron Disease are rapidly progressive and fatal neurodegenerative diseases that affect people in their prime. Poor understanding of the processes that lead to these diseases have slowed drug development. Through innovative experimental design, we aim to decipher a novel disease mechanism that involves specific molecular interactions and translate these findings into new therapies for the diseases.
Repeat Expansions In Neurological Disease: Discovery, Interpretation And Enhanced Diagnostics
Funder
National Health and Medical Research Council
Funding Amount
$889,937.00
Summary
Identifying the mutation or genetic cause of disease in an individual is the first step in the provision of appropriate clinical care and treatment. This diagnostic process is being revolutionised through the ability to sequence the entire human genome in a time and cost effective manner. This project will enable identification of novel and known repeat expansion using whole genome sequencing, providing rapid diagnoses and better clinical care for individuals with neurogenetic disorders.
New Therapeutic Approaches For Genetic Skeletal Disorders
Funder
National Health and Medical Research Council
Funding Amount
$961,150.00
Summary
Genetic skeletal disorders are a significant disease burden with limited therapeutic options. We will use mouse models of cartilage and bone disorders and human induced pluripotent stem cell derived bone and cartilage 'disease in a dish' models to test repurposed FDA-approved drugs and new drug candidates as novel therapeutic strategies.