Splice Correction As A Treatment For Rare Diseases
Funder
National Health and Medical Research Council
Funding Amount
$824,316.00
Summary
We propose that a strategy of bringing effective and personalised treatments to amenable patients with rare genetic diseases, though ambitious, is readily achievable and opportune. Importantly, a consensus approach will facilitate expediting potentially curative treatments for many patients with rare diseases that would be unlikely to be commercially viable if considered individually.
Molecular Imaging Of Cancers With Copper And Zirconium Radiopharmaceuticals
Funder
National Health and Medical Research Council
Funding Amount
$466,475.00
Summary
This research aims to develop new imaging agents to assist in the diagnosis of breast cancer. This research will advance knowledge in the areas of chemistry, biotechnology and diagnostic imaging.
A New Strategy To Prevent Anthracycline-induced Cardiotoxicity While Improving Anti-cancer Activity
Funder
National Health and Medical Research Council
Funding Amount
$318,034.00
Summary
The anthracycline-based drugs such as doxorubicin currently used for cancer treatment have a major side effect in that they induce heart damage. We have shown that doxorubicin action can be modified to result in greater tumour cell kill, but also with reduced death of cardiac cells. We now aim to develop a molecular understanding of this process in order to allow better design of chemotherapy regimes that include the anthracyclines.
Novel Radioligands For PET Imaging And Phenotyping Of Multiple Sclerosis Diagnosis
Funder
National Health and Medical Research Council
Funding Amount
$817,828.00
Summary
This project aims to catalyse a paradigm shift in the management of multiple sclerosis (MS). We will achieve this paradigm shift by developing radioligands that can be used to image the brain of MS patients. These agents will target a protein in MS patients that will be diagnostic of the state of disease progression. This will facilitate prediction of disease outcome, monitoring of therapeutic response and the development of novel treatment strategies for the better treatment of MS.
Discovery Of New Tuberculosis Drug Leads Targeting Cell Wall Biosynthesis
Funder
National Health and Medical Research Council
Funding Amount
$714,816.00
Summary
There is a desperate need for the development of new therapies for the treatment of TB due to widespread resistance of Mycobacterium tuberculosis, the causative agent of TB, to current therapies. The overall goal of this research project is to identify new TB drug leads through the development of structural analogues of bacterially-derived natural products called the sansanmycins that inhibit cell wall synthesis in Mycobacterium tuberculosis.
Targeted Alternative Splicing: A Common Therapeutic Platform To Treat Inherited Diseases
Funder
National Health and Medical Research Council
Funding Amount
$797,165.00
Summary
Genes consist of exons (protein coding domains) separated by introns (non-coding intervening sequences). It is now evident that not every exon need be included in the gene message to direct synthesis of a functional gene product. This application seeks to identify which exons are essential for gene function and those exons that are "dispensable". Such information will allow personalized therapies to be developed based on splice switching, as we have done for Duchenne muscular dystrophy.