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
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.
ARC Research Network in Spatially Integrated Social Science. The ARC Research Network in Spatially Integrated Social Science (SISS) builds Australia's capacity and capability for innovative, collaborative, cross-disciplinary effort to investigate the impacts of change on the behaviour and well-being of people and the fortunes of places. SISS theories and research tools permit the integration of diverse and complex databases, the generation of new synthetic datasets, the incorporation of spatial ....ARC Research Network in Spatially Integrated Social Science. The ARC Research Network in Spatially Integrated Social Science (SISS) builds Australia's capacity and capability for innovative, collaborative, cross-disciplinary effort to investigate the impacts of change on the behaviour and well-being of people and the fortunes of places. SISS theories and research tools permit the integration of diverse and complex databases, the generation of new synthetic datasets, the incorporation of spatial concepts into statistical analysis and modelling, powerful visualisation of information, and the building spatial decision support systems, to provide an improved evidence base and better informed decision-making to address the significant challenges facing Australia's people and its places.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
Industrial Transformation Training Centres - Grant ID: IC170100016
Funder
Australian Research Council
Funding Amount
$3,123,492.00
Summary
ARC Training Centre for Personalised Therapeutics Technologies. The ARC Training Centre for Personalised Therapeutics Technologies aims to create and develop the skills and technology to benefit from the transformative impacts that cell/organ-on-a-chip technology will have on the medtech/pharma industries. By combining microfluidics-based/real-time technologies with personalised medicine the Training Centre will provide industry growth opportunities through improved screening of potential therap ....ARC Training Centre for Personalised Therapeutics Technologies. The ARC Training Centre for Personalised Therapeutics Technologies aims to create and develop the skills and technology to benefit from the transformative impacts that cell/organ-on-a-chip technology will have on the medtech/pharma industries. By combining microfluidics-based/real-time technologies with personalised medicine the Training Centre will provide industry growth opportunities through improved screening of potential therapeutics. The use of an individual patient’s cellular and molecular research findings will ultimately enable personalised diagnostic and therapeutic decisions.Read moreRead less
Ethical and Social Issues in Translating Epigenetics into Antenatal Care. This project aims to investigate how knowledge about the intergenerational transmission of health and disease is shaping antenatal care in Australia. It expects to generate new knowledge about how the science of epigenetics can be used to address social inequality and is anticipated to have impact across the social sciences, bioethics and public health. Expected outcomes of the project include novel theoretical approaches ....Ethical and Social Issues in Translating Epigenetics into Antenatal Care. This project aims to investigate how knowledge about the intergenerational transmission of health and disease is shaping antenatal care in Australia. It expects to generate new knowledge about how the science of epigenetics can be used to address social inequality and is anticipated to have impact across the social sciences, bioethics and public health. Expected outcomes of the project include novel theoretical approaches to the ethics of pregnancy, along with guidance tools to shape the use of epigenetics in antenatal care and social policy to reduce social inequalities. This should provide significant benefits, improving information and support available to vulnerable women as they negotiate maternal health and social services. Read moreRead less
Testing co-evolutionary processes driving venom diversity in tiger snakes. Testing co-evolutionary processes driving venom diversity in tiger snakes. This project aims to examine the geographic variation amongst tiger snakes in anatomy, ecology, and life history traits, and the relationship of these factors to venom toxins and production; and to evaluate the true pharmacological potential of tiger snake venom. This project will investigate the role of venom adaptation in long-term animal evoluti ....Testing co-evolutionary processes driving venom diversity in tiger snakes. Testing co-evolutionary processes driving venom diversity in tiger snakes. This project aims to examine the geographic variation amongst tiger snakes in anatomy, ecology, and life history traits, and the relationship of these factors to venom toxins and production; and to evaluate the true pharmacological potential of tiger snake venom. This project will investigate the role of venom adaptation in long-term animal evolution, by identifying rare venom transcripts involved in providing evolutionary potential for adaptation to environmental change. This is essential as continuing climatic and human-induced alteration of our environment affects southern Australia where many people live, work and interact with native wildlife. Anticipated outcomes are maximizing venom harvests and enhanced snakebite treatment capacity.Read moreRead less
Work, care, retirement and health: ageing agendas. This project will undertake a gendered analysis of how Australians can retire well, taking account of their key resources (such as work, superannuation, the aged pension, and other assets) and key demands (such as to work longer and to care for others in the context of an ageing population and a more feminised workforce).
Molecular Characterisation Of Early Precursor Lesions Of A Novel Ñserrated Pathwayî Of Colorectal Cancer Using Gene Expression And Proteomics.
Funder
National Health and Medical Research Council
Funding Amount
$318,338.00
Summary
In Australia, CRC is the second highest cause of all cancer-related deaths. If detected early, CRC has a high success rate of cure, but a percentage of precursor lesions escape detection and show aggressive clinical behaviour to progress to CRC. These are difficult to diagnosis with existing technologies. We aim to understand the biology behind sessile serrated adenoma pathways and hence enhance early detection, diagnosis and treatments strategies.