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
Special Research Initiatives - Grant ID: SR0354908
Funder
Australian Research Council
Funding Amount
$10,000.00
Summary
The Insect-Plant Chemical Ecology Network (IPCEN). We bring together plant molecular biology, entomology and analytical chemistry to transform three leading fields of Australian research into an advanced science with far reaching capabilities in innovative research and applied outcomes. Expertise studying the biochemical pathways that produce specific plant compounds and expertise in insect recognition and response to these chemicals will be brought together. This will lead to new research outco ....The Insect-Plant Chemical Ecology Network (IPCEN). We bring together plant molecular biology, entomology and analytical chemistry to transform three leading fields of Australian research into an advanced science with far reaching capabilities in innovative research and applied outcomes. Expertise studying the biochemical pathways that produce specific plant compounds and expertise in insect recognition and response to these chemicals will be brought together. This will lead to new research outcomes and solutions to problems in agriculture, horticulture, forestry and protection of Australia's native flora. Researchers are struggling to create these links, constrained by disciplinary boundaries and geographical isolation. Key industries and researchers already support this proposal.Read moreRead less
Prevalence report by the Australian Advisory Board on Autism Spectrum Disorders (ASD) estimated that 1 child in every 160 children in the 6-12 year-old age group is affected by ASD. There is no cure for ASD and the causes are not understood. We propose that sex hormones may play a role in the development of these disorders. We will test this hypothesis using knockout and transgenic mouse models which have social interaction deficits and brain structure reminiscent of these disorders.
ARC Centre of Excellence in Plants for Space. ARC Centre of Excellence in Plants for Space. This Centre aims to create on-demand, zero-waste, high-efficiency plants and plant products to address grand challenges in sustainability for Space and on Earth. Significant advances in plant, food, and sensory science; process and systems engineering; law and policy; and psychology are expected to deliver transformative solutions for Space habitation – and create enhanced plant-derived food and bioresour ....ARC Centre of Excellence in Plants for Space. ARC Centre of Excellence in Plants for Space. This Centre aims to create on-demand, zero-waste, high-efficiency plants and plant products to address grand challenges in sustainability for Space and on Earth. Significant advances in plant, food, and sensory science; process and systems engineering; law and policy; and psychology are expected to deliver transformative solutions for Space habitation – and create enhanced plant-derived food and bioresources to capitalise upon emergent and rapidly expanding domestic and global markets. Anticipated outcomes include industry uptake of innovative plant forms, foods, technologies, and commodities; and an ambitious education and international co-ordination agenda to position Australia as a global leader in research supporting Space habitation.Read moreRead less