Gene Discovery And Functional Insights For Neurological And Retinal Disorders
Funder
National Health and Medical Research Council
Funding Amount
$2,163,220.00
Summary
Understanding the genetic drivers of disease is key for the development of disease therapies. Determination of the causal genetic variants in a disorder can be used for future diagnosis, prognostication, and personalised treatment. We have previously identified ~20 novel genes and developed new methods providing genomic diagnoses for 1000s of individuals. In the next five years I will make significant advances in our understanding of what causes diseases such as epilepsy, ataxia and dementia.
Saving Sight Through Novel BioTech Innovations For Inherited Retinal Disease
Funder
National Health and Medical Research Council
Funding Amount
$1,534,523.00
Summary
Inherited retinal diseases (IRDs) are the leading cause of blindness in working-aged adults. My work focuses on learning more about IRDs, including the correlation between genes and the degenerative changes in the eye. I am developing new outcome measures to predict who might lose vision faster, and to use in clinical trials. I will then use this knowledge in two clinical trials for new IRD treatments - a gene therapy and an electronic device that is implanted into the eye.
Computational Modelling To Understand Early-stage Neurodegeneration
Funder
National Health and Medical Research Council
Funding Amount
$645,205.00
Summary
Rather than attempting to reverse neurodegeneration, therapeutic strategies must target the earliest possible stages of disease, when treatments have the potential to prevent or slow down pathological progression. The proposed project will employ computational modelling using functional MRI to deliver highly efficient and sensitive markers of Familial Alzheimer’s disease and Huntington’s disease progression to inform when in the progression of disease clinical trials should take place.
Faecal Microbiota Transplantation And Other Novel Therapeutic Microbial Manipulation Strategies In Inflammatory Bowel Disease
Funder
National Health and Medical Research Council
Funding Amount
$645,205.00
Summary
There is growing interest in the role of microbial-based strategies including faecal microbiota transplantation (FMT) for the treatment of inflammatory bowel disease. This project will develop such strategies into valid treatment options through a combination of clinical & basic science work including (1) characterising viral & fungal factors of importance, (2) evaluation of novel orally-delivered formulations of FMT, and (3) development of better defined, more reproducible microbial treatments.
The Role Of A Low Emulsifier Diet To Treat Crohn's Disease
Funder
National Health and Medical Research Council
Funding Amount
$447,603.00
Summary
Currently, there are no specific diets known to treat Crohn’s disease (CD). Initial lab- and animal-based studies have indicated emulsifiers added to food drive inflammation in CD, but no human trials completed. This study will design and feed high and low emulsifier diets to healthy and CD subjects and measure markers of inflammation. This will establish the role of a low emulsifier diet to treat CD.
Improving Patient Outcomes In Familial Hypercholesterolaemia Across The Continuum Of Care
Funder
National Health and Medical Research Council
Funding Amount
$645,205.00
Summary
'Familial' means it runs in families and 'hypercholesterolaemia' means high blood cholesterol. Familial Hypercholesterolaemia (FH) is the most common and serious form of inherited high cholesterol. People with FH are at an increased risk of developing cardiovascular disease. High cholesterol also placed an estimated $4 billion burden on the Australian economy in 2017-18. Practical research on prevention strategies will improve cardiovascular health outcomes and lower the burden of health costs.
Novel Approaches To Nanomedicines For Future Therapies
Funder
National Health and Medical Research Council
Funding Amount
$2,414,215.00
Summary
Nanomedicines have the potential to transform healthcare by targeting significant health issues such as Alzheimer’s, diabetes and Parkinson’s diseases that have mainly eluded successful therapeutic solutions. In addition, nanotechnology has the potential to significantly improve the treatment of chronic pain by repurposing analgesic medications for improved effectiveness without significant side effects. I will target these two areas of research during the next five years.
Understanding Cell Signalling As A Basis For New Therapeutics
Funder
National Health and Medical Research Council
Funding Amount
$2,231,372.00
Summary
This Investigator grant will capitalise on my extensive expertise in determining the three-dimensional atomic structures of proteins to uncover fundamental biological mechanisms in cancer and Alzheimer’s disease as a basis for discovering new drugs to combat these devastating diseases.
Novel Nanotechnology Strategies For Drug Co-delivery And Combined Therapies In The Brain
Funder
National Health and Medical Research Council
Funding Amount
$1,512,250.00
Summary
Key challenges for treating brain diseases include effective delivery of drugs into the brain and targeted delivery to pathogenic areas. I have developed two world-first drug delivery systems that address these challenges. This project will expand their loading and brain delivery capability to deliver a broad range of novel multiple therapeutics to target sites in the brain. Human brain disease models will be used for systematic preclinical evaluation of novel delivery systems and therapeutics.
Elucidating The Pathological Role And Predictive Value Of Mental Health Disorder Risk Genes
Funder
National Health and Medical Research Council
Funding Amount
$1,562,250.00
Summary
Mental health disorders such as schizophrenia and depression are common and often debilitating conditions. The genes in our DNA play a large role in who develops these disorders and many risk genes have been identified. We will investigate when and how these risk genes are “switched on” to work out how they cause disease and to accurately predict who is at high risk of developing a mental health disorder. These advances will help us to understand disease causation and to improve treatments.