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
Development of cloning technology for the Australian Pig Industry. Cloning has the potential to be the most efficient of the reproductive technologies developed for increasing genetic improvement in livestock. Currently up to 5% of cloned embryos develop to term in the pig. This is higher than that reported for cattle and sheep. Moreover the use of this technology in the pig does not appear not to result in the same sorts of problems and losses seen around the time of birth in these species ....Development of cloning technology for the Australian Pig Industry. Cloning has the potential to be the most efficient of the reproductive technologies developed for increasing genetic improvement in livestock. Currently up to 5% of cloned embryos develop to term in the pig. This is higher than that reported for cattle and sheep. Moreover the use of this technology in the pig does not appear not to result in the same sorts of problems and losses seen around the time of birth in these species i.e. the majority of cloned pigs appear normal and are healthy at birth. However before cloning can be used commercially, current efficiencies need to be increased approx two fold for this to be economically viable. The aim of the present study is to improve the efficiency of our current cloning protocol and develop associated technologies such as embryo freezing to facilitate commercialisation. This will ensure that the Australian Pig Industry remains competitive at a pivotal time in its development.Read moreRead less
Enhancing fertility for the Thoroughbred and Standardbred industries. Enhancing fertility for the Thoroughbred and Standardbred industries. Responding to industry calls for improved methods of detecting and managing infertility in both stallions and mares, this project aims to provide a platform for competitive collaborations between universities, biotechnology companies and horse breeders: the end-users of technological developments in equine reproduction. The Thoroughbred and Standardbred bree ....Enhancing fertility for the Thoroughbred and Standardbred industries. Enhancing fertility for the Thoroughbred and Standardbred industries. Responding to industry calls for improved methods of detecting and managing infertility in both stallions and mares, this project aims to provide a platform for competitive collaborations between universities, biotechnology companies and horse breeders: the end-users of technological developments in equine reproduction. The Thoroughbred and Standardbred breeding industries contribute over $6.5 billion per annum to the Australian economy and employ thousands of staff across their value chains. However, these industries suffer from breeding program inefficiencies that amount to over $800 million in annual losses. Expected outcomes are novel reproductive technologies and specialised research capabilities that will make the Australian equine industry a global leader.Read moreRead less
Frontotemporal Dementia And Motor Neurodegenerative Syndromes
Funder
National Health and Medical Research Council
Funding Amount
$11,583,107.00
Summary
Frontotemporal degeneration of the brain is a leading cause of morbidity. It is a pathologically heterogeneous group of rapidly-progressive disorders with behavioural, language and motor deficits. This research program brings together international leaders in clinical, pathological and biological research of these syndromes, aiming to fast track new knowledge and innovations to develop the necessary tools and therapies to effectively diagnose, manage and treat these disorders.
Cyclostome bryozoans: Their use as paleoceanographic, paleoclimatologic and paleoenvironmental proxies throughout the cenozoic of southern Australia. The unravelling of ocean evolution relies largely on data from fossils - geochemical proxies of ancient oceans. Southern Australia contains an inrivalled 45 million-year old record of cool-water carbonate sedimentation and thus global paleoceanography. Currently, the only proxies available in these extensive sediments are brachiopods and planktic f ....Cyclostome bryozoans: Their use as paleoceanographic, paleoclimatologic and paleoenvironmental proxies throughout the cenozoic of southern Australia. The unravelling of ocean evolution relies largely on data from fossils - geochemical proxies of ancient oceans. Southern Australia contains an inrivalled 45 million-year old record of cool-water carbonate sedimentation and thus global paleoceanography. Currently, the only proxies available in these extensive sediments are brachiopods and planktic foraminfers. However, these sediments contain prolific low-magnesium calcite bryozoans, particularly cyclostomes, that could be used instead. This research will assess such bryozoans and test their usefulness and reliability, utilising existing extensive modern marine and Tertiary samples, data bases and sections. Such potential proxies would be globally useful for limestones from the last 500My.Read moreRead less
Effects Of Latrepirdine On Beta Amyloid Clearance, Aggregation And Neurodegeneration In Alzheimer�s Disease
Funder
National Health and Medical Research Council
Funding Amount
$512,647.00
Summary
Alzheimer's disease (AD) is becoming more common with our growing aged population and currently no treatment exists that halts disease progress. The increasing health costs of AD underscore the need for development of any treatment that will slow or halt AD pathogenesis. By understanding the mechanisms of action of a drug [latrepirdine] that has recently shown some promise in phase II clinical trials, related drugs that are more specific and potent will be developed.
Enhancing the efficiency of equine reproduction: relevant to the Thoroughbred and Standardbred breeding industries. The purpose of this project is to link the equine breeding industry with a major centre of reproductive research at the University of Newcastle. By creating this unique nexus, major efficiency gains will be achieved for this industry that will not only secure its international competitiveness but also significantly enhance its profitability.
Molecular and genetic analysis of epigenetic components in a model plant. Australia is a major exporter of agricultural food crops thus producers must maintain their competitive advantage in order to compete on the world stage. Food crops unfortunately have large, complex genomes that are not sequenced and a generation time of months that makes research outcomes slow to achieve. This project proposes to utilise a model plant that has a small completely sequenced genome and a short generation tim ....Molecular and genetic analysis of epigenetic components in a model plant. Australia is a major exporter of agricultural food crops thus producers must maintain their competitive advantage in order to compete on the world stage. Food crops unfortunately have large, complex genomes that are not sequenced and a generation time of months that makes research outcomes slow to achieve. This project proposes to utilise a model plant that has a small completely sequenced genome and a short generation time making it ideal to study the fundamental biological process of RNA silencing. Discoveries and outcomes from this project may have the potential to benefit Australian crops, ecosystems and human health.Read moreRead less
Digestive Physiology of Crocodilians: Towards an Improved Diet and Feeding Regime for Use by the Farming Industry. Crocodile farming promotes the sustainable production and consumption of crocodile products without harming natural populations. The industry grosses over $15 million per year in Australia and $200 million worldwide. Costs associated with transporting and storing food are the single largest expense that crocodile farms have. Increases in the efficiency of converting food into growth ....Digestive Physiology of Crocodilians: Towards an Improved Diet and Feeding Regime for Use by the Farming Industry. Crocodile farming promotes the sustainable production and consumption of crocodile products without harming natural populations. The industry grosses over $15 million per year in Australia and $200 million worldwide. Costs associated with transporting and storing food are the single largest expense that crocodile farms have. Increases in the efficiency of converting food into growth will have positive economic benefits to the industry. Efficiencies can be produced by (1) increasing food absorption, (2) decreasing the energetic costs of digestion, and (3) producing a new food that is less expensive to ship and store. This project will directly address the first two of these and will lay the foundation for the development of the third. Read moreRead less