Targeting The Oncoprotein MDMX As A Novel Treatment For Triple Negative Breast Cancer
Funder
National Health and Medical Research Council
Funding Amount
$561,672.00
Summary
Breast cancer (BrCa) is a leading cause of cancer death in women worldwide. BrCas unable to respond to current therapies have the worst outcomes. We propose a novel strategy to treat these cancers, based on our new findings. Our two protein targets are: (1) MDMX, that we found drives BrCa with its partner, (2) mutant p53, which causes cancer spread. We plan to directly target these drivers of aggressive BrCas, using new drugs that individually show great promise in trials in a number of cance
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.
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.
Blood-Brain Barrier Penetrating Antisense Therapy For Spinal Muscular Atrophy
Funder
National Health and Medical Research Council
Funding Amount
$635,005.00
Summary
Spinal muscular atrophy (SMA) is a genetic disease caused by the deficiency of a protein known as survival motor neuron.This results in the degeneration of motor neurons (nerve cells controlling muscles) leading to progressive muscle weakness, paralysis, and eventual death. Currently, there is no known cure for SMA. The aim of proposed research is to develop gene-modifying molecules that prevent degeneration of motor neuron and extend the life-span of mice as a potential therapy for SMA.
Optimization Of Splice Switching Therapies To Treat Duchenne Muscular Dystrophy
Funder
National Health and Medical Research Council
Funding Amount
$448,827.00
Summary
Duchenne muscular dystrophy, the most common and serious form of childhood muscle wasting, is caused by mutations in the dystrophin gene that block synthesis of the normal product. Antisense oligomers have been used in clinical trials to remove the disease-causing part of the message and rescue expression. Clinical trials have demonstrated proof-of-concept, although individual responses varied. This application seeks to improve the therapeutic potential of these compounds.