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.
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.
HARMONIC ANALYSIS AND BOUNDARY VALUE PROBLEMS FOR ELLIPTIC SYSTEMS. It is of the utmost necessity for Australia to develop the theoretical
expertise needed in the current era. The type of mathematics under
investigation here is closely allied to that needed in much of the
current boom in communication technology and medical research. The
training which would be provided to the research associates is
considerable, and would flow on to produce the expertise needed to
keep the coming gen ....HARMONIC ANALYSIS AND BOUNDARY VALUE PROBLEMS FOR ELLIPTIC SYSTEMS. It is of the utmost necessity for Australia to develop the theoretical
expertise needed in the current era. The type of mathematics under
investigation here is closely allied to that needed in much of the
current boom in communication technology and medical research. The
training which would be provided to the research associates is
considerable, and would flow on to produce the expertise needed to
keep the coming generation involved in modern technological development. I will maintain my large collaborative effort with
leading mathematicians from the US, France and other countries, thus
helping to keep Australia at the forefront of a significant field of
research.Read moreRead less
HARMONIC ANALYSIS OF ELLIPTIC SYSTEMS ON RIEMANNIAN MANIFOLDS. It is of the utmost necessity for Australia to develop the theoretical expertise needed in the current era. The type of mathematics under investigation here is closely allied to that needed in much of the current boom in communication technology and medical research. The training which would be provided to the research associates is considerable, and would flow on to produce the expertise needed to keep the coming generation invol ....HARMONIC ANALYSIS OF ELLIPTIC SYSTEMS ON RIEMANNIAN MANIFOLDS. It is of the utmost necessity for Australia to develop the theoretical expertise needed in the current era. The type of mathematics under investigation here is closely allied to that needed in much of the current boom in communication technology and medical research. The training which would be provided to the research associates is considerable, and would flow on to produce the expertise needed to keep the coming generation involved in modern technological development. I will maintain my active collaborative effort with leading mathematicians from the US, France and other countries, thus helping to keep Australia at the forefront of a significant field of research.Read moreRead less
Propagation of singularities for the Schrodinger equation. The time-dependent Schrodinger equation governs the evolution of quantum particles. In this project we aim to use new techniques from mathematical scattering theory to analyse solutions of the Schrodinger equation and obtain sharp bounds on their singularities. Controlling such singularities will allow us to deduce quantitative bounds on the number of eigenvalues in certain situations, and provide new techniques for studying nonlinear Sc ....Propagation of singularities for the Schrodinger equation. The time-dependent Schrodinger equation governs the evolution of quantum particles. In this project we aim to use new techniques from mathematical scattering theory to analyse solutions of the Schrodinger equation and obtain sharp bounds on their singularities. Controlling such singularities will allow us to deduce quantitative bounds on the number of eigenvalues in certain situations, and provide new techniques for studying nonlinear Schrodinger equations. Read moreRead less
Physics of Risk: new tools to survey the Australian market and beyond. The lives of most Australians depend on the dynamics of financial markets that affects investments, savings, business, employment, growth, wealth and -ultimately- the daily functioning of our society. Understanding, monitoring and managing the dynamics of financial markets is of crucial importance to policy-makers, financial institutions and businesses that are increasingly faced with managing risk, planning strategies and ta ....Physics of Risk: new tools to survey the Australian market and beyond. The lives of most Australians depend on the dynamics of financial markets that affects investments, savings, business, employment, growth, wealth and -ultimately- the daily functioning of our society. Understanding, monitoring and managing the dynamics of financial markets is of crucial importance to policy-makers, financial institutions and businesses that are increasingly faced with managing risk, planning strategies and taking decisions in an increasingly complex market-place. The project is also of importance to the continued evolution of physics in this country contributing to the emergence of a strong new area of statistical physics concerned with the ?real world? in a manner hitherto unknown.Read moreRead less
HARMONIC ANALYSIS, BOUNDARY VALUE PROBLEMS, AND MAXWELL'S EQUATIONS IN LIPSCHITZ DOMAINS. Boundary value problems for partial differential equations arise naturally when physical problems are expressed in mathematical terms. This project concerns the systematic development of the harmonic analysis of partial differential operators, and of the corresponding boundary integrals in order to solve such problems on irregular regions. Particular emphasis is given to studying the behaviour of electrom ....HARMONIC ANALYSIS, BOUNDARY VALUE PROBLEMS, AND MAXWELL'S EQUATIONS IN LIPSCHITZ DOMAINS. Boundary value problems for partial differential equations arise naturally when physical problems are expressed in mathematical terms. This project concerns the systematic development of the harmonic analysis of partial differential operators, and of the corresponding boundary integrals in order to solve such problems on irregular regions. Particular emphasis is given to studying the behaviour of electromagnetic waves both inside and outside irregularly shaped surfaces, and their propagation through it.Read moreRead less
Harmonic analysis of differential operators in Banach spaces. This proposal aims to develop harmonic analysis (the mathematical tools used in digital music and photography) in new contexts. It focuses on boundary value problems (the theory behind medical or geological imaging) and stochastic equations (which describe phenomena with random components such as the behaviour of financial markets).