Harnessing RNA Interference In Gene Therapy Vectors For ?-thalassaemia
Funder
National Health and Medical Research Council
Funding Amount
$719,188.00
Summary
There is an urgent need to develop safe and effective treatments for ?-thalassaemia. We anticipate that ?-globin-specific RNAi sequences will synergise with ?-globin transgene expression to achieve balanced ?-/?-globin ratio in a clinical setting. Given that one of the major issues with current gene therapy vectors is achieving high levels of expression, we believe this will be a more effective gene therapy strategy than ?-globin transgene expression alone.
Characterisation Of CBF Acute Myeloid Leukaemia By MicroRNA Profiling
Funder
National Health and Medical Research Council
Funding Amount
$118,956.00
Summary
Recent studies have demonstrated the existence of small pieces of previously undescribed genetic material, known as microRNAs (miRNAs), which are thought to have critical functions across various biological processes and regulatory pathways in cells. This project aims to examine the role of these miRNAs in the development of abnormal cellular proliferation that leads to leukaemia, by examining the expression of all known miRNAs in the abnormal cells of our patients with leukaemia.
Epigenetic Regulation Of Self Renewal And Lineage Commitment In Haematopoiesis
Funder
National Health and Medical Research Council
Funding Amount
$1,104,930.00
Summary
The process by which all our mature blood cells are produced and sustained remains largely unknown. Underpinning the cell fate decisions made through blood cell development is the tightly regulated expression of key genes and proteins that subsequently direct the process of blood cell differentiation. This project will aim study and uncover the molecular mechanisms that coordinate the key gene expression programs that lead to normal blood cell development.
Defining The Role Of RNA Editing In Erythropoiesis
Funder
National Health and Medical Research Council
Funding Amount
$628,945.00
Summary
We are seeking to understand how red blood cells are produced. We have identified that a process called RNA editing may be important in the regulating the production of red blood cells.
In Australia seventeen persons are diagnosed with leukemia or a related blood disorder each day. An understanding of the way the body controls blood cell production has widespread relevance to diseases like leukemia and the way that they are treated. The burden of this disease on the Australian health budget will continue to increase dramatically as our population ages in coming years. Moreover, an understanding of blood cell production has wider relevance to similar control mechanisms in other ....In Australia seventeen persons are diagnosed with leukemia or a related blood disorder each day. An understanding of the way the body controls blood cell production has widespread relevance to diseases like leukemia and the way that they are treated. The burden of this disease on the Australian health budget will continue to increase dramatically as our population ages in coming years. Moreover, an understanding of blood cell production has wider relevance to similar control mechanisms in other organs, as nature reuses its best inventions. Australia has had an enviable international record in discoveries related to factors controlling blood cells and we wish to continue that tradition by examining an exciting new development involving microRNAs. These molecules are found in all normal cells but they have been ignored until recently. Although the field is in its infancy, we now know that these microRNAs are likely to be involved in many cellular processes. In this project we will study the importance of this new class of regulatory molecule in order to discover previously-hidden functions in normal blood cells and their malignant counterparts (leukemias) in humans. The team comprises two laboratories which have collaborated for many years and already a number of exciting molecular methods have been developed. Ultimately this project may lead to novel treatments involving gene therapy, bone marrow transplantation and blood hormones.Read moreRead less