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
Advanced materials for space propulsion: satellites and cubesats. Poorly controlled interactions between plasmas and surfaces often mean loss of process efficiency and surface degradation over time. For Hall thrusters, a type of engine used to move satellites in space, this means increased fuel consumption and shorter useful life. Through modelling and experiment, this project will show how intelligent selection of advanced materials and plasma parameters can minimise surface wear, enable in sit ....Advanced materials for space propulsion: satellites and cubesats. Poorly controlled interactions between plasmas and surfaces often mean loss of process efficiency and surface degradation over time. For Hall thrusters, a type of engine used to move satellites in space, this means increased fuel consumption and shorter useful life. Through modelling and experiment, this project will show how intelligent selection of advanced materials and plasma parameters can minimise surface wear, enable in situ material repair to extend device lifetime, and modulate plasma properties to increase thruster efficiency for a given task. These benefits enable reliable propulsion platforms for massive communication and observation satellite networks and deep space exploration.Read moreRead less
Towards a steady-state fusion reactor: understanding and controlling eruptive instabilities in tokamaks. The lifetime of future fusion reactors may be severely compromised by periodic releases of internal energy which can damage the reactor walls. In an international research effort that is based on unique Australian optical technologies, the project aims to resolve some of the key issues that should allow these instabilities to be suppressed.
Discovering And Targeting Genes Regulating Skeletal Muscle Function, Metabolism, And Adaptations To Exercise Interventions
Funder
National Health and Medical Research Council
Funding Amount
$431,000.00
Summary
Muscle wasting and decreased in mitochondrial function due to ageing or lack of physical activity are associated with reduced quality of life. The overarching aim is to develop a unique research program focusing on targeting specific genes, and to discover novel genes regulating muscle wasting and mitochondrial (dis)function. I anticipate this approach to assist in the development of targeted and personalised prevention and therapy for diseases associated with muscle (dis)function.
Structural And Functional Analysis Of A Cancer-linked Co-regulator Complex
Funder
National Health and Medical Research Council
Funding Amount
$729,571.00
Summary
We seek to understand the mechanisms by which genes are switched on and off throughout our lifetime. A number of multi-component protein machines are involved in this process but their make-up and mechanism of action is not understood. We will investigate the structure and function of one of these machines that has been strongly linked to cancer.
Understanding helium induced nanostructure formation. This project addresses the interaction dynamics of high-flux helium particles with materials that drives surface nanowire growth. These dynamics are important to nuclear reactor materials and to developing new nanotechnology materials for high energy density lithium-ion battery anodes and water splitting catalysts. Through model and experiment, this project expects to generate new knowledge of processes that drive sub-surface nano-bubble form ....Understanding helium induced nanostructure formation. This project addresses the interaction dynamics of high-flux helium particles with materials that drives surface nanowire growth. These dynamics are important to nuclear reactor materials and to developing new nanotechnology materials for high energy density lithium-ion battery anodes and water splitting catalysts. Through model and experiment, this project expects to generate new knowledge of processes that drive sub-surface nano-bubble formation and surface nanowire growth in materials exposed to helium particles. This project will result in improved understanding of material degradation during nuclear reactor operation and will make a new contribution to high-value manufacturing capabilities for next generation energy systems.Read moreRead less
Linkage Infrastructure, Equipment And Facilities - Grant ID: LE120100021
Funder
Australian Research Council
Funding Amount
$150,000.00
Summary
A diagnostics platform for advanced plasma-chemical analysis. A wide range of production processes involve the use of plasmas to modify materials, but they are not well understood. This project will give Australian researchers the tools to look inside plasma processes and fully characterise them for the first time, unlocking new knowledge and providing new insight into the plasma processing environment.
Understanding molecular negative ion production for use in pathology. The project aims to increase the yield of molecular negative ion sources by improving our understanding of the formation of ion beams from plasma sources and expand our knowledge of molecular negative ion generation in plasma environments leading to brighter ion beams. For example, understanding cancer requires cellular level tools to map how cells are changing. These maps are made using ion beams which are scanned across cell ....Understanding molecular negative ion production for use in pathology. The project aims to increase the yield of molecular negative ion sources by improving our understanding of the formation of ion beams from plasma sources and expand our knowledge of molecular negative ion generation in plasma environments leading to brighter ion beams. For example, understanding cancer requires cellular level tools to map how cells are changing. These maps are made using ion beams which are scanned across cells to remove material that is analysed at the atomic and molecular level. Ion beams are produced from plasma sources, but much of their operation is not understood. Such improved ion beams are expected to enable inexpensive and fast cellular level pathology at even small hospitals to tackle cancer for society’s benefit.Read moreRead less
Physics of a new low power electrothermal radiofrequency plasma thruster. Electric propulsion is the new wave of attitude control for spacecraft. Space engines must be small, lightweight and able to run unattended for over 20 years in a very harsh environment. The physics of a new electrothermal radiofrequency plasma thruster will be investigated. Neutral gas heating will be initially quantified by optical spectroscopy combined with computer generated simulated spectra. A space ready prototype w ....Physics of a new low power electrothermal radiofrequency plasma thruster. Electric propulsion is the new wave of attitude control for spacecraft. Space engines must be small, lightweight and able to run unattended for over 20 years in a very harsh environment. The physics of a new electrothermal radiofrequency plasma thruster will be investigated. Neutral gas heating will be initially quantified by optical spectroscopy combined with computer generated simulated spectra. A space ready prototype will be designed, manufactured and developed to carry out direct measurements of thrust and gas heating in our large space simulation vacuum facility.Read moreRead less
Plasma-soft tissue interactions: advancing a new era of plasma technologies. This project aims to develop the prerequisite knowledge to underpin new plasma technologies. Specifically, the project plans to provide new insight into the interaction between ionised gas plasma jets and biological soft tissue. It plans to use advanced analytical techniques and modelling simulations to investigate the physics of the plasma jet and processes occurring at the jet–tissue interface. It also plans to use (b ....Plasma-soft tissue interactions: advancing a new era of plasma technologies. This project aims to develop the prerequisite knowledge to underpin new plasma technologies. Specifically, the project plans to provide new insight into the interaction between ionised gas plasma jets and biological soft tissue. It plans to use advanced analytical techniques and modelling simulations to investigate the physics of the plasma jet and processes occurring at the jet–tissue interface. It also plans to use (bio)engineered tissue targets, chemical, biological and cellular assays to probe the transport and effects of plasma reactive species deep within tissue. This new knowledge would enable the development of new plasma sources and protocols for applications in health, medicine, biotechnology and manufacturing.Read moreRead less