Gene Therapy Research Unit

Microscope image of liver cells expressing a green fluorescent protein







When biomedical knowledge is undergoing rapid growth (as it is at present), the gap between our capacity to diagnose and our capacity to treat disease widens.  For this reason it is important to increase our efforts to realise the therapeutic potential of advances in biological knowledge.  Such efforts demand that we explore novel therapeutic approaches and surmount the formidable basic science and translational challenges inherent in taking progress in the laboratory through to effective therapy.

With the potential to correct the genetic basis of disease, gene therapy (the use of genes as medicine) represents one of the most exciting prospects in medical research.  The road to success, however, is long and difficult, and much basic pre-clinical and clinical research is required.

The Gene Therapy Research Unit, a joint initiative of the Children’s Medical Research Institute and The Children’s Hospital at Westmead, was established in recognition of the exciting potential of gene therapy and the need for a research environment that combines excellence in both basic science and clinical care.  The research activities of the Unit reflect the challenges outlined above.

For more information see the staff profiles and projects pages.