Development Of Therapeutically Useful Human Artificial Chromosomes For Gene Delivery And Optimal Gene Expression
Funder
National Health and Medical Research Council
Funding Amount
$496,986.00
Summary
Gene therapy is an exciting new form of treatment for genetic disorders aimed at providing long-term correction of the problems at source - namely the affected gene. The biggest technical hurdle facing gene therapy is to be able to deliver the therapeutic genes efficiently and safely into patient cells. Many gene therapy protocols are currently being trialled clinically. These protocols, based mostly on the use of attenuated viruses to deliver the genes, carry potential risks to the patients in ....Gene therapy is an exciting new form of treatment for genetic disorders aimed at providing long-term correction of the problems at source - namely the affected gene. The biggest technical hurdle facing gene therapy is to be able to deliver the therapeutic genes efficiently and safely into patient cells. Many gene therapy protocols are currently being trialled clinically. These protocols, based mostly on the use of attenuated viruses to deliver the genes, carry potential risks to the patients in terms of infection, immune response, and germline modification. We have developed the first stage of a new technology for gene delivery that does not require the use of viruses. This technology is based on the generation of human artificial chromosomes, which are smaller versions of the naturally occurring chromosomes that carry all the genes inside our cells. Safety in these artificial chromosomes comes from the use of entirely human materials for their engineering. These artificial chromosomes also have other advantages over the viral approaches, including allowing large genes to be carried, and providing a permanent cure in a single treatment. We have already successfully constructed, published, and patented a number of first-generation human artificial chromosomes. The current project aims to complete the next proof-of-concept milestone towards the further development of this technology. Specifically, we propose to demonstrate the ability of the artificial chromosomes to carry genes and provide sustainable expression of these genes in cells and in animal models. Success in this study will allow the technology to proceed rapidly into commercialisation and clinical trial as a new improved tool for gene delivery and gene therapy.Read moreRead less
Eddy-resolving global ocean-sea ice modelling. Eddy-resolving global ocean-sea ice modelling. This project aims to develop a world-class global ocean-sea ice model framework through a nationwide consortium. The resulting high resolution models are expected to provide the foundation for the next decade of Australian ocean-sea ice modelling capacity. This research should lead to improved ocean and sea ice prediction, ocean reanalyses, and climate projections, enhancing Australia's capacity to pred ....Eddy-resolving global ocean-sea ice modelling. Eddy-resolving global ocean-sea ice modelling. This project aims to develop a world-class global ocean-sea ice model framework through a nationwide consortium. The resulting high resolution models are expected to provide the foundation for the next decade of Australian ocean-sea ice modelling capacity. This research should lead to improved ocean and sea ice prediction, ocean reanalyses, and climate projections, enhancing Australia's capacity to predict the ocean state on timescales of days to decades. This is expected to yield efficiencies in shipping, marine search and rescue and naval operations, and increase the accuracy of projected future changes in climate, sea level, ocean ecosystems and the cryosphere.Read moreRead less
We have discovered a single tumour factor which causes cancer cachexia, a wasting condition that is one of the worst complications of malignancy, for which there is no current effective treatment. We have developed antibodies which effectively block this condition in preclinical models and have produced human/humanised version of this. This application is to characterise these human antibodies to allow us proceed to clinical trials.
Helping Central Banks Measure Unobserved Variables Using Real-time Forecasts. The project addresses structural measurement problems confronted routinely by central bankers. The techniques developed, and the estimates provided, will aid directly the Partner Organisations (the Reserve Bank of Australia, the Reserve Bank of New Zealand and Norges Bank) and other central banks in formulating monetary policy. The analysis will allow interest rates in Australia and elsewhere to be set with greater pre ....Helping Central Banks Measure Unobserved Variables Using Real-time Forecasts. The project addresses structural measurement problems confronted routinely by central bankers. The techniques developed, and the estimates provided, will aid directly the Partner Organisations (the Reserve Bank of Australia, the Reserve Bank of New Zealand and Norges Bank) and other central banks in formulating monetary policy. The analysis will allow interest rates in Australia and elsewhere to be set with greater precision. The techniques developed in this project will facilitate the understanding and communication of monetary policy within the central banks concerned, and enhance communication of monetary policy strategy to the public.Read moreRead less
Coupling tropical cyclone and climate physics with ocean waves. It is argued that without accounting for the wave effects directly, the physics of large-scale air-sea interactions is inaccurate and incomplete. The project will introduce explicit coupling of large-scale atmospheric and oceanic phenomena with the physics of surface waves which should lead to improved predictions of tropical cyclones and climate.
Epigenetic Programming Of Immune Development In Utero: Role Of The Maternal Environment In The Allergy Epidemic
Funder
National Health and Medical Research Council
Funding Amount
$764,463.00
Summary
This study will provide new insights into the development of allergic disease. Specifically, we will explore the hypothesis that allergic disease and other disorders or immune dysregulation occur as a result of gene-environmental interactions in early life, and that these events begin in pregnancy when the developing fetus is still developing and most susceptible to these effects.
Evaluating the weather in climate models - the relationship of dynamics and rainfall over Australia in current and future climates. Climate change will be experienced by society as a change in the day-to-day weather. This project will investigate the capabilities of modern climate models in simulating the weather with a particular focus on rainfall, and will provide guidance to the use of these models in projections of the future of Australia's climate.
ARC Centres of Excellence for Climate System Science. Our capacity to assess the threat of climate change is undermined by an unacceptable level of uncertainty in the understanding and modelling of regional climates. The Centre will undertake world-class research targeting identified weaknesses in the physical, chemical and biological components of the climate system. We will engage and nurture graduate students and postdoctoral follows through a program of graduate training and mentoring to per ....ARC Centres of Excellence for Climate System Science. Our capacity to assess the threat of climate change is undermined by an unacceptable level of uncertainty in the understanding and modelling of regional climates. The Centre will undertake world-class research targeting identified weaknesses in the physical, chemical and biological components of the climate system. We will engage and nurture graduate students and postdoctoral follows through a program of graduate training and mentoring to permanently transform our understanding of climate systems science particularly for the Australian region. The key outcome will be a dramatic enhancement in national capacity to understand and project the scale of future regional climate change.Read moreRead less
Cloudiness over the Southern Ocean: reducing a key knowledge gap and source of climate model uncertainty. Southern Ocean clouds are key ingredients of the global climate system and yet are only poorly understood and poorly represented in climate models. Through the use of advanced observational analysis techniques this research will provide a deep understanding of key Southern Ocean cloud regimes and improve their representation in models.
Global trends in oceanic wind speed and wave height. This project will determine whether winds and waves over the world's oceans have changed over the past 30 years. Such information is critically important in understanding global climate change, evaporation, air-sea interaction and to safely design and operate coastal and offshore facilities.