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Research Topic : GENE THERAPY
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  • Funded Activity

    Novel Therapeutic Strategies To Reduce The Burden Of Chronic Heart Failure

    Funder
    National Health and Medical Research Council
    Funding Amount
    $4,928,323.00
    Summary
    The broad aims of the Program are to develop novel strategies in the prevention and treatment of chronic heart failure. This will involve investigating new targets for pharmacological therapies, evaluating whether common co-morbid disease states such as diabetes alter the efficacy of these therapies and investigating the role of stem-cell therapy in this setting. The Program will also evaluate the contribution of non-heart failure drugs to the burden of heart failure, determine the impact of rur .... The broad aims of the Program are to develop novel strategies in the prevention and treatment of chronic heart failure. This will involve investigating new targets for pharmacological therapies, evaluating whether common co-morbid disease states such as diabetes alter the efficacy of these therapies and investigating the role of stem-cell therapy in this setting. The Program will also evaluate the contribution of non-heart failure drugs to the burden of heart failure, determine the impact of rurality on prescribing for this condition and explore systems of optimising delivery of best practice to the community. This research formalises the existing collaborative efforts of a team of investigators that span all aspects of research into the therapeutics of CHF from basic laboratory research to evaluation of patients in clinical trials and public health translational aspects of this condition. The Chief Investigators and Principal Investigators have an existing successful research collaboration which will be greatly expanded via Program.
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    Funded Activity

    Molecular Determinants Of Risk, Progression And Treatement Response In Melenoma

    Funder
    National Health and Medical Research Council
    Funding Amount
    $12,947,193.00
    Summary
    Melanoma is a major Australian health problem. It is the third most common cancer in men and women and has a disproportionately heavy impact on productive years of life because it is the common cause of cancer death in younger adults. The investigators are all associated with the Melanoma Institute Australia, incorporating the Sydney Melanoma Unit (SMU). MIA is the world’s largest clinical service dedicated to the treatment of melanoma, treating >1500 new melanoma patients annually and mainta .... Melanoma is a major Australian health problem. It is the third most common cancer in men and women and has a disproportionately heavy impact on productive years of life because it is the common cause of cancer death in younger adults. The investigators are all associated with the Melanoma Institute Australia, incorporating the Sydney Melanoma Unit (SMU). MIA is the world’s largest clinical service dedicated to the treatment of melanoma, treating >1500 new melanoma patients annually and maintains a repository of clinical data on melanoma and a large melanoma tissue bank. The Program has also recruited large numbers of people from the community, as well as people with a strong family history of melanoma, in order to study its causes. It aims to utilise these internationally-recognised resources to develop a scientific basis for 1) improved management of individuals at high risk for development and progression of melanoma, and 2) improved treatment of patients with early and disseminated melanoma, in an era of rapid change in the prospects of successfully treating this dangerous cancer. The Program will do this by consolidating and extending its existing collaborative research, supported by NHMRC since 2006.
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    Funded Activity

    Translational Research In Stroke

    Funder
    National Health and Medical Research Council
    Funding Amount
    $4,652,613.00
    Summary
    Many new therapies are being developed to treat stroke and prevent its recurrence. While a number of these have been successfully introduced, there is a puzzling gap between evidence of efficacy in animal models and humans. One of the main reasons may be lack of an integrated approach between the basic sciences and clinical researchers. By assembling a team with skills in both areas and a structure to maximise communication between groups this team plan to incrementally introduce new therapies i .... Many new therapies are being developed to treat stroke and prevent its recurrence. While a number of these have been successfully introduced, there is a puzzling gap between evidence of efficacy in animal models and humans. One of the main reasons may be lack of an integrated approach between the basic sciences and clinical researchers. By assembling a team with skills in both areas and a structure to maximise communication between groups this team plan to incrementally introduce new therapies into clinical practice.
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    Funded Activity

    Regulation Of Neural Cell Production In The Normal And Diseased Brain

    Funder
    National Health and Medical Research Council
    Funding Amount
    $6,888,658.00
    Summary
    Members of this team are at the forefront of research into the molecular control of nerve cell production and function in the developing and adult brain. They were responsible, often through collaboration, for many of the major discoveries demonstrating that stem cells in the brain of adult animals can generate new nerve cells; this revolutionised our concept of the brain and opened-up the possibility of therapeutic repair of neural damage through stimulation of a patient?s own stem cells. Disco .... Members of this team are at the forefront of research into the molecular control of nerve cell production and function in the developing and adult brain. They were responsible, often through collaboration, for many of the major discoveries demonstrating that stem cells in the brain of adult animals can generate new nerve cells; this revolutionised our concept of the brain and opened-up the possibility of therapeutic repair of neural damage through stimulation of a patient?s own stem cells. Discovering the molecular mechanisms controlling this process is the goal of the present program. In order to achieve this aim we have formed a team on the basis of considerable past success, as well as future requirements. The team members have the complementary skills to assess all aspects of the problem, including crucial functional and clinical expertise. By combining resources we will position ourselves at the very forefront of the international competition to discover and to evaluate clinically the molecular mechanisms underlying neural repair and regeneration. This is of enormous significance in determining how we best treat stroke, injury and other neurodegenerative diseases in the next 10 years, and will lead to the development of new therapeutics of immense value. The team will use innovative approaches such as cell-sorting to obtain pure populations of stem cells and their progeny in order to identify new therapeutic targets; these will then be validated in animal models of neurological disease. Since team members have previously been involved in progressing molecular discovery to clinical trials, we are also in a good position to exploit these discoveries in partnership with the biopharmaceutical industry.
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    Funded Activity

    Genetic And Bioinformatic Analysis Of Complex Human Diseases

    Funder
    National Health and Medical Research Council
    Funding Amount
    $8,752,567.00
    Summary
    Some human diseases are common in families; examples include prostate cancer, blood cancers, epilepsy and diabetes. Therefore, close relatives of individuals with a disease have an increased risk of being affected by this disease, implying a genetic basis. Finding the cause of these diseases is difficult, we will be developing novel approaches to the identification of genes responsible for these diseases. This is the first step towards the development of treatments for affected individuals.
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    Funded Activity

    Posttraumatic Mental Health: Enhancing Resilience And Recovery

    Funder
    National Health and Medical Research Council
    Funding Amount
    $5,043,011.00
    Summary
    Psychological disorders following exposure to trauma account for a significant proportion of the burden of disease in terms of personal suffering, decreased productivity, occupational and social dysfunction, medical disorders, and demands on health services. The overall goal of this project is to enhance the nation’s capacity in research into the identification of risk factors for posttraumatic mental disorders, study of neurophysiological factors mediating these disorders, and evaluation of tre .... Psychological disorders following exposure to trauma account for a significant proportion of the burden of disease in terms of personal suffering, decreased productivity, occupational and social dysfunction, medical disorders, and demands on health services. The overall goal of this project is to enhance the nation’s capacity in research into the identification of risk factors for posttraumatic mental disorders, study of neurophysiological factors mediating these disorders, and evaluation of treatment strategies to reduce psychological morbidity after trauma. This project will develop a critical mass of Australia’s leading trauma researchers that will ensure that Australia retains its leading edge in posttraumatic research.
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    Funded Activity

    Immune Regulation, Effector Function And Therapy

    Funder
    National Health and Medical Research Council
    Funding Amount
    $5,323,077.00
    Summary
    The immune system plays an important role in protecting the host from viral and bacterial infections, and inhibits cancer onset and progression. Unfortunately the immune system can sometimes lose specificity and attack the host resulting in autoimmune diseases such as diabetes. This research team has played a vital role in characterising the specific activities of immune cells and the associated factors. By understanding these complex processes the team aims to harness the unique therapeutic pro .... The immune system plays an important role in protecting the host from viral and bacterial infections, and inhibits cancer onset and progression. Unfortunately the immune system can sometimes lose specificity and attack the host resulting in autoimmune diseases such as diabetes. This research team has played a vital role in characterising the specific activities of immune cells and the associated factors. By understanding these complex processes the team aims to harness the unique therapeutic properties of our own immune system and translate their findings into the clinic.
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    Funded Activity

    The Biological Function Of Genes On Human Chromosome 21 And Their Role In The Pathophysiology Of Down Syndrome

    Funder
    National Health and Medical Research Council
    Funding Amount
    $2,821,602.00
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    Funded Activity

    Leucocyte & Endthelial Cell Biology

    Funder
    National Health and Medical Research Council
    Funding Amount
    $4,928,323.00
    Summary
    The foot soldiers of the immune system, the white blood cells, constantly march through the body seeking out invaders, but kept in check by the barrier of endothelial cells that lines the inside of blood vessels. When infection occurs, molecular messages are transmitted amongst the white cells and between white cells and edothelium, to activate the immune cells to pass out of the blood vessels and mount a defence. Unfortunatley, the activation system sometimes goes awry, resulting in inflammator .... The foot soldiers of the immune system, the white blood cells, constantly march through the body seeking out invaders, but kept in check by the barrier of endothelial cells that lines the inside of blood vessels. When infection occurs, molecular messages are transmitted amongst the white cells and between white cells and edothelium, to activate the immune cells to pass out of the blood vessels and mount a defence. Unfortunatley, the activation system sometimes goes awry, resulting in inflammatory or allergic disease, such as arthritis or asthma. This team of researchers from the Hanson Institute in Adelaide, combining expertise in molecular and cell biology, protein chemestry, structual biology and animal models, has been working together for over 10 years, investigating the molecular mechanisms involved in controlling the formation and activities of blood vessels and white blood cells. This program seeks to further that understanding, and to develop drugs that have the potential of ameliorating the inflammatory condition.
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    Funded Activity

    Regulation Of Gene Expression: Biomolecular Interactions In Cellular Development And Disease

    Funder
    National Health and Medical Research Council
    Funding Amount
    $2,998,713.00
    Summary
    This team consists of three of Australia�s younger researchers Merlin Crossley, Joel Mackay and Jacqui Matthews (as Chief Investigators), who are recognized as authorities in the areas of gene regulation and the structural and functional analysis of proteins. They are joined by Mitchell Weiss, a world authority on blood development and clinical disorders,and Alexis Verger, a molecular and cell biologist recruited from France, both as Principal Investigators. Crossley, Mackay and Matthews have wo .... This team consists of three of Australia�s younger researchers Merlin Crossley, Joel Mackay and Jacqui Matthews (as Chief Investigators), who are recognized as authorities in the areas of gene regulation and the structural and functional analysis of proteins. They are joined by Mitchell Weiss, a world authority on blood development and clinical disorders,and Alexis Verger, a molecular and cell biologist recruited from France, both as Principal Investigators. Crossley, Mackay and Matthews have worked as a team for around six years to date, have published together in high-quality international journals, and have received anumber of accolades for their contributions to Australian science. For example, Crossley has won a number of national awards, including the Gottschalk Medal of the Australian Academy of Science; Mackay was recently awarded the Prime Minister�s Prize for Life Scientist of the Year, and Matthews won the only Charles and Sylvia Viertel Medical Research Fellowship to be awarded in 2003. The members of this team have collaborated extensively on the world stage and Crossley, Mackay and Matthews have also taken leadership roles in the Australian scientific community. Mitchell Weiss has been an important collaborator, exchanging reagents and advice, since he and Crossley trained together as postdocs in Stu Orkin�s lab at Harvard in the early 90s. Most recently Weiss, in collaboration with Mackay, has made important discoveries on a-globin production, which has led to several highly significant publications including a seminal paper in Cell in 2004.The program of research put forward in this proposal centres around understanding the mechanisms through which genes are switched on and off, using blood development as a model system, that is also fundamental to human life. The regulation of gene output is essential both during the development of an organism and throughout the course of its life. Problems with this regulation can result in many different disease states, most notably cancer, which includes the many different types of leukemias. At one level, gene output is controlled by networks of specific proteins known as transcription factors that interact both with each other and with DNA. Currently, however, the details surrounding which complexes regulate which genes and the processes that control the making and breaking up of the complexes are not well understood. Knowledge of how these interactions take place will put us in a position to control the output of chosen genes for therapeutic purposes. We propose to use a combination of cell biological, biochemical, and structural approaches to firstly shed light on these complexes and secondly develop reagents that can be used to manipulate the activity of specific genes.
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