Developing Exon Replacement Gene Therapy To Cure Rett Syndrome: An Innovative Model For Neurodevelopmental Disorders
Funder
National Health and Medical Research Council
Funding Amount
$475,105.00
Summary
There is no cure for neurodevelopmental disorders such as Rett syndrome which is caused by mutations in the MECP2 gene. Gene therapy is ineffective due to the 'Goldilocks' effect where too little, as well as too much expression of MECP2 causes disease. Here a gene editing therapy will be tested in patient cells and mouse model that will maintain the normal cellular expression of MECP2 by cutting out the mutated regions of the gene (exons) and replacing them with repaired copies.
Vascular Changes Are A Key Contributor To And Novel Drug Target For Interferon-alpha Induced Neurological Disease
Funder
National Health and Medical Research Council
Funding Amount
$1,245,401.00
Summary
Type I interferons (IFN-Is) contribute to wide range of neurological diseases including ageing and neurodegeneration. At its extreme IFN-I-mediated neurodegeneration is known as 'interferonopathy'. The mechanisms of how IFN-Is drive disease are unclear, making causal treatment difficult. We have recently uncovered ground-breaking evidence that abnormal blood vessels are a key contributor to the disease. Here, we will investigate novel treatment targets for patients with interferonopathies.
Targeting The NLRP3 Inflammasome And Interleukin-18 In Hypertensive Heart Failure
Funder
National Health and Medical Research Council
Funding Amount
$1,241,115.00
Summary
Heart failure is a common complication of hypertension and a major cause of death and disability worldwide. This project will characterise a newly identified inflammatory pathway that we believe to be a major cause of the enlargement and scarring of the heart that accompanies hypertension. We will also trial drugs that block this inflammatory pathway to determine their suitability as future therapies for this devastating disease.
Silencing Pulmonary Nociceptors To Treat Severe Respiratory Viral Infections
Funder
National Health and Medical Research Council
Funding Amount
$534,173.00
Summary
The lungs receive a rich supply of nerve fibres, many of which play an important role in helping defend against pathogens, including viruses. When viral infections become severe, too much inflammation occurs in the lungs and this creates a serious and difficult to treat clinical problem. Hundreds of thousands of people each year die from the complications of severe lung infections. We are investigating a potential new therapy that targets the lung nerves and relieves excessive inflammation.
Innate Threat Detection Circuits In The Superior Colliculus Co-ordinate Respiratory And Cardiovascular Responses To Visual Stimuli
Funder
National Health and Medical Research Council
Funding Amount
$517,958.00
Summary
Our surroundings affect our bodies: light pollution, traffic, and aircraft noise all significantly affect cardiovascular health. This project will investigate interactions between brain systems that subconsciously scan our surroundings for interesting or threatening features, and those that co-ordinate the cardiovascular and respiratory systems. We will generate new knowledge that describes how the brain detects danger and translates this into signals that contribute to cardiovascular risk.
Influenza A Viral Infection And Pregnancy Complications
Funder
National Health and Medical Research Council
Funding Amount
$1,346,858.00
Summary
Pregnant women who contract influenza are 5 times more likely to be hospitalised than the general population. Babies of mothers with influenza are also associated with increased perinatal mortality rates. We hypothesise that influenza infection in pregnancy significantly impairs the maternal vascular system resulting in maternal and foetal morbidity. Outcomes from this research may change current treatment modalities to improve maternal and foetal outcomes complicated by influenza infection.