Can Exercise Early After Spinal Cord Injury Prevent Deterioration Of Muscle And Bone?
Funder
National Health and Medical Research Council
Funding Amount
$775,049.00
Summary
Spinal cord injury leads to a profound deterioration of the muscles and bones in the paralysed limbs. This project will examine the effects of exercising the paralysed limbs as early as possible after injury to prevent muscle and bone loss rather than restoring the tissues once changes have occurred. The time course and mechanisms underlying the microstructural decay of bone over the first year after injury will also be examined to provide a basis for determining fracture risk in this group.
Advancing Glycine To The Clinic For Duchenne Muscular Dystrophy
Funder
National Health and Medical Research Council
Funding Amount
$248,978.00
Summary
We have identified the therapeutic potential of the amino acid glycine for Duchenne muscular dystrophy (DMD), the most common and severe of the muscular dystrophies. To facilitate rapid translation to the clinic, this proposal will; 1) examine the effect of glycine on lifespan and quality of life in mouse models of DMD; 2) determine glycine’s mode of action; and 3) investigate whether these effects represent further benefits to those currently used gold standard treatments.
Cancer cachexia is a devastating disease characterised by muscle wasting, weakness and fatigue. It impairs patient quality of life and accounts for >20% of cancer-related deaths. This project will identify factors responsible for cancer cachexia and develop new strategies to alleviate wasting and weakness in cancer patients, to improve their quality of life and reduce mortality.
Modelling Leber’s Hereditary Optic Neuropathy Using Human Induced Pluripotent Stem Cells
Funder
National Health and Medical Research Council
Funding Amount
$628,416.00
Summary
Leber’s Hereditary Optic Neuropathy (LHON) is a blinding disease that affects young males and is caused by the death of cells in the optic nerve. To better understand LHON, this project utilises induced pluripotent stem (iPS) cells for disease modelling. iPS cells will be generated from patients and turned into optic nerve cells, allowing us to study the diseased cells in the laboratory, providing a platform to screen for novel drugs to improve treatment options and fast-track drug development.
Therapeutic Potential Of Modulating Heat Shock Protein Expression For Muscle Wasting Disorder
Funder
National Health and Medical Research Council
Funding Amount
$1,172,146.00
Summary
Heat shock proteins help stressed proteins fold back to their original conformation and restore function. In a discovery published in Nature we identified induction of heat shock protein 72 (Hsp72) as a novel approach for muscular dystrophy and other conditions where there is inflammation and muscle weakness. This proposal will investigate whether Hsp72 induction is similarly effective in tackling the muscle wasting and weakness in conditions like ageing and frailty and in muscle injury.
OXPHOS Upregulation To Preserve Vision In Leber's Hereditary Optic Neuropathy
Funder
National Health and Medical Research Council
Funding Amount
$496,874.00
Summary
Leber's Hereditary Optic Neuropathy (LHON) is a devastating blinding disease that preferentially affects young men. Sufferers have normal vision until teenage years or their twenties when a rapid loss of vision occurs that results in permanent blindness. It is caused by genetic changes in the mitochondrial DNA that we inherit from our mothers. The mitochondria are the cells' energy generators. We aim to use molecules similar to female hormones to boost energy as a new treatment to preserve visio ....Leber's Hereditary Optic Neuropathy (LHON) is a devastating blinding disease that preferentially affects young men. Sufferers have normal vision until teenage years or their twenties when a rapid loss of vision occurs that results in permanent blindness. It is caused by genetic changes in the mitochondrial DNA that we inherit from our mothers. The mitochondria are the cells' energy generators. We aim to use molecules similar to female hormones to boost energy as a new treatment to preserve vision in at-risk LHON individuals.Read moreRead less
We have designed novel gene therapy agents to treat MND. This therapeutic approach uniquely combines gene therapy agents with antibodies which stimulate motor neuron health and connections to muscle. This project will comprehensively characterise the therapeutic effects of our novel gene therapy agents in MND mice. We predict that our gene therapy complexes will improve motor neuron survival,motor function and lifespan in MND mice.
Role Of ABCA8 Transporter In Oligodendroglial Lipid Regulation And Multiple System Atrophy
Funder
National Health and Medical Research Council
Funding Amount
$651,516.00
Summary
Multiple system atrophy (MSA) is a rapid-onset brain disorder impacting on multiple functions of the body resulting in death. The cause of MSA is unknown and there is no cure. In MSA brains, the oligodendroglial cells are impaired and cannot properly make myelin (specialized lipid membrane), which is required for the proper functioning of the nerve cells in the brain. The aim of this project is to find out how changes in lipid in the brain impact on the MSA disease process.