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
DROP DEFORMATION IN CONFINED MICROFLUIDIC GEOMETRIES. Increasingly, high technology applications in biotechnology and microtechnology industries need to process complex (non-Newtonian) fluids with dispersed particles/droplets in channels as small as several microns (microfluidics). A computational fluid dynamic model of non-Newtonian droplet deformation in microfluidic geometries will be developed, and validated using experimental measurements of the flow field in this project. The aim is to und ....DROP DEFORMATION IN CONFINED MICROFLUIDIC GEOMETRIES. Increasingly, high technology applications in biotechnology and microtechnology industries need to process complex (non-Newtonian) fluids with dispersed particles/droplets in channels as small as several microns (microfluidics). A computational fluid dynamic model of non-Newtonian droplet deformation in microfluidic geometries will be developed, and validated using experimental measurements of the flow field in this project. The aim is to understand and quantify factors influencing droplet deformation. Coupling non-Newtonian characteristics with microfluidic geometries will allow the continuous manufacture of micro-particles of specified size and shape for existing and new applications, and will provide guidance for further extending the process to nano-particle manufacture.Read moreRead less
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.
Industrial Transformation Training Centres - Grant ID: IC170100032
Funder
Australian Research Council
Funding Amount
$4,272,072.00
Summary
ARC Training Centre in Fire Retardant Materials and Safety Technologies. The ARC Training Centre in Fire Retardant Materials and Safety Technologies aims to train a cohort of industry-focused researchers to improve the fire safety of lightweight materials and structures and fire protection systems. The Training Centre expects to create knowledge on novel green and durable fire retardant materials, advanced fire models for urban and built environment, fire suppression technologies, and new flamma ....ARC Training Centre in Fire Retardant Materials and Safety Technologies. The ARC Training Centre in Fire Retardant Materials and Safety Technologies aims to train a cohort of industry-focused researchers to improve the fire safety of lightweight materials and structures and fire protection systems. The Training Centre expects to create knowledge on novel green and durable fire retardant materials, advanced fire models for urban and built environment, fire suppression technologies, and new flammability tests for compliance with fire safety regulatory standards. An expected outcome of this Training Centre is to accelerate the transformation of Australia’s industries in fire retardant materials, products and engineering services.Read moreRead less
Enabling low greenhouse gas emissions from road vehicles through the proper use of alternative fuels. A major increase in alternative transport fuel use appears necessary in our response to the challenges of climate change and energy security. This proposal will advance our fundamental understanding of key aspects of the combustion of particular alternative fuels, thus enabling proper engine design and so maximising greenhouse and energy security benefits. Further, the Australian automotive indu ....Enabling low greenhouse gas emissions from road vehicles through the proper use of alternative fuels. A major increase in alternative transport fuel use appears necessary in our response to the challenges of climate change and energy security. This proposal will advance our fundamental understanding of key aspects of the combustion of particular alternative fuels, thus enabling proper engine design and so maximising greenhouse and energy security benefits. Further, the Australian automotive industry is a major employer and exporter, and needs to develop and/or maintain international leadership in low emission technologies to ensure its long term viability. This proposal builds a consortium of local organisations with common interests, thus helping local industry respond to several, significant challenges that they presently face.Read moreRead less
Thermal transport in multi-phase flows for concentrating solar applications. This project seeks to advance the field of heat transfer in high-temperature systems involving liquid metals, with emphasis on energy storage and solar power technologies. The concept couples a tubular sodium boiler with a sodium chloride phase-change storage system for continuous energy supply. Sodium chloride is low cost and has a melting temperature suitable for a wide range of industrial processes. The project plans ....Thermal transport in multi-phase flows for concentrating solar applications. This project seeks to advance the field of heat transfer in high-temperature systems involving liquid metals, with emphasis on energy storage and solar power technologies. The concept couples a tubular sodium boiler with a sodium chloride phase-change storage system for continuous energy supply. Sodium chloride is low cost and has a melting temperature suitable for a wide range of industrial processes. The project plans to address the challenge of sodium stability in highly irradiated tubes by investigating mass, momentum, energy and radiative transport in liquid metals. It is intended that this will inform the design and testing of novel sodium boilers to provide stable and isothermal process heat for continuous or on-demand production of power, chemical fuels and commodities.Read moreRead less
Thermal transport by design for fast and efficient solar thermochemical fuel production. This project aims to demonstrate the utility of the thermal transport by design approach to develop functionally graded reactive materials that allow for fast and efficient solar thermo-chemical fuel production. Prediction capabilities will be developed to optimise multi-scale radiative and gas transport coupled with non-stoichiometric redox reactions. Synthesis gas production will be demonstrated using the ....Thermal transport by design for fast and efficient solar thermochemical fuel production. This project aims to demonstrate the utility of the thermal transport by design approach to develop functionally graded reactive materials that allow for fast and efficient solar thermo-chemical fuel production. Prediction capabilities will be developed to optimise multi-scale radiative and gas transport coupled with non-stoichiometric redox reactions. Synthesis gas production will be demonstrated using the new structures in a prototype solar thermochemical reactor under high-flux irradiation. This project aims to advance the fields of thermal sciences and high-temperature solar thermochemical processing and expand the engineering knowledge base to pave the way to sustainable transportation with the existing infrastructure.Read moreRead less
ARC Centre for Kangaroo Genome. In this Australian-led Kangaroo Genome Project, we will map and characterize the tammar wallaby genome at the molecular level. Marsupial genomes are uniquely valuable because they provide comparisons that reveal new human genes, regulatory sequences and marsupial-specific genes. These will deliver new products and information useful for medicine, industry, agriculture and conservation. We will construct integrated genetic and physical maps of the genome, clone the ....ARC Centre for Kangaroo Genome. In this Australian-led Kangaroo Genome Project, we will map and characterize the tammar wallaby genome at the molecular level. Marsupial genomes are uniquely valuable because they provide comparisons that reveal new human genes, regulatory sequences and marsupial-specific genes. These will deliver new products and information useful for medicine, industry, agriculture and conservation. We will construct integrated genetic and physical maps of the genome, clone the whole genome as large inserts in BAC vectors, and build a "golden path" with minimal overlap. We will construct libraries of expressed genes from tammar tissues and array them for use in analysing gene expression.Read moreRead less