summary

We are investigating possible ways of treating genetic disorders. One method involves gene therapy - introducing 'healthy' copies of genes into a patient's cells. This concept has proved harder to implement than previously thought. For example, the large size of most human genes has necessitated the use of 'stripped-down' versions of these genes. However, minimising the amount of genetic material used can exclude stretches of DNA that would normally control the gene's function.

Safer and more efficient ways of delivering these 'replacement' genes directly to their target cells need to be devised. Research is also required on how to keep the inserted DNA intact and retain its normal functions in the cell. We are very aware of the serious concerns about the safety and effectiveness of gene therapy, and are committed to addressing these issues.

We are also investigating treatments based on cell therapy, and the use of drugs to modify gene expression. In many genetic illnesses, it may even be possible to alter other genes pharmacologically so as to overcome the disease.

Dr Jim Vadolas Cell & Gene Therapy Murdoch Childrens Research Institute Royal Children's Hospital Flemington Road Parkville Victoria 3052 Australia

T +61 3 8341 6232 (Office) T +61 3 8341 6236 (Lab) F +61 3 9348 1391 E jim.vadolas@mcri.edu.au

Group Leader Biography

Dr Jim Vadolas completed his PhD at the Departments of Microbiology and Immunology, University of Melbourne, and postdoctoral training at the Murdoch Childrens Research Institutewith Panos Ioannou and Bob Williamson. In 2005, Jim became group leader of the Cell and Gene Therapy group at the MCRI.He is primarily interested in the development ofnew therapeutic strategies for thalassaemia and related haemoglobinopathies. His work has led the establishment of several new model systems that can be used to identify and evaluate potential therapies. He is currently an Executive Committee member of the Australasian Gene Therapy Society. Jim is also an Executive Committee Member of Thalassaemia Australia.

Project 1: Development of RNAi therapy for thalassaemia

-Thalassaemia is an inherited disease caused by defective synthesis of the -globin chain of haemoglobin, leading to imbalanced globin chains. Excess -chains precipitate in erythroid progenitor cells resulting in cell death, ineffective erythropoiesis and severe anaemia. Decreased -globin chain synthesis leads to milder symptoms, exemplified by individuals who co-inherit -thalassaemia and -thalassaemia. Therefore, a possible therapeutic strategy in the treatment of -thalassaemia could include targeted reduction of -globin chains to mimic co-inheritance of /-thalassaemia. One way of reducing -chain synthesis is by using RNA interfering (RNAi). Numerous studies have shown promising results utilising RNAi in vitro and in vivo. This study will investigate the use of RNAi-mediated reduction of -globin chains for the therapy for -thalassaemia.

Originally posted here:
Murdoch Childrens Research Institute : cell & gene therapy

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