Improving The Safety Characteristics Of Lentiviral Vectors.
Funder
National Health and Medical Research Council
Funding Amount
$296,250.00
Summary
Gene therapy holds great promise for the treatment of many types of disease including inherited disorders, cancer and cardiovascular disorders. However, the potential of gene therapy has in many cases been limited by the lack of suitable technologies for gene delivery. We have developed a novel gene delivery vehicle from human immunodeficiency virus type 1 (HIV-1). Although this vehicle has many of the characteristics desired of a gene therapy vector its derivation from a retrovirus, particularl ....Gene therapy holds great promise for the treatment of many types of disease including inherited disorders, cancer and cardiovascular disorders. However, the potential of gene therapy has in many cases been limited by the lack of suitable technologies for gene delivery. We have developed a novel gene delivery vehicle from human immunodeficiency virus type 1 (HIV-1). Although this vehicle has many of the characteristics desired of a gene therapy vector its derivation from a retrovirus, particularly one with such an unenviable reputation, raises obvious safety concerns. In order to properly address this issue it is necessary that the vector is carefully designed and properly tested. This project aims to continue our rational, systematic and stepwise approach to the development of our vector with the aim of producing a vector that can be used with a high degree of confidence in its safety, such that it is suitable for clinical usage. Given the highly desirable properties of these vectors, and the wide range of diseases where their use is being considered, the availability of such a vector will have great significance for the widespread practical application of gene therapy. Indeed, several of the projects we are developing with our vector will in all likelihood lead to lead to clinical trials and it is clear that the conduct of these trials will depend on the availability of a suitable vector.Read moreRead less
I am a developmental biologist using genetic approaches in the model vertebrate, zebrafish, to study the molecular basis for muscle formation. My laboratory studies the basic biology of muscle and applies that knowledge to create accurate disease models o
Studies Of Myeloid Leukaemogenesis In The Zebrafish
Funder
National Health and Medical Research Council
Funding Amount
$171,827.00
Summary
This project aims to investigate the causes of white blood cell cancer, or leukaemia, at the molecular level, using a novel approach in zebrafish. Zebrafish provide a powerful experimental model for developmental genetics, largely due to the visual and technical accessibility of embryos for experimentation. We plan to introduce a fluorescent molecular tag into the white blood cells in order to directly visualise them. We will then predispose these fish to leukaemia and screen for mutants with en ....This project aims to investigate the causes of white blood cell cancer, or leukaemia, at the molecular level, using a novel approach in zebrafish. Zebrafish provide a powerful experimental model for developmental genetics, largely due to the visual and technical accessibility of embryos for experimentation. We plan to introduce a fluorescent molecular tag into the white blood cells in order to directly visualise them. We will then predispose these fish to leukaemia and screen for mutants with enhanced or suppressed leukaemia. We anticipate that the mutants will allow new genes involved in the development of leukaemia to be identified.Read moreRead less
Production Of Humanised Mouse Models For Haemoglobin E And 0-thalassaemia
Funder
National Health and Medical Research Council
Funding Amount
$280,693.00
Summary
The proposed study aims to identify and characterise genes critical to male fertility using two mouse models of infertility: 1) Joey mouse line: an ENU induced model of sperm abnormalities. Following linkage analysis, candidate genes will be selected for sequencing to identify the causal mutation. 2) Ggn knockout mice. The role of the testis-specific gene, Ggn will be characterised through a phenotypic analysis of Ggn knockout mice and a series of expression and biochemical analyses. Both models ....The proposed study aims to identify and characterise genes critical to male fertility using two mouse models of infertility: 1) Joey mouse line: an ENU induced model of sperm abnormalities. Following linkage analysis, candidate genes will be selected for sequencing to identify the causal mutation. 2) Ggn knockout mice. The role of the testis-specific gene, Ggn will be characterised through a phenotypic analysis of Ggn knockout mice and a series of expression and biochemical analyses. Both models will be of direct value in the identification of commercially relevant contraceptive targets, as well as furthering our understanding of male reproductive function.Read moreRead less