Gene Therapy Research Unit

A joint initiative of The Children's Hospital at Westmead and Children's Medical Research Institute

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Introduction

The ultimate aim of biomedical research is to improve human health, but its more immediate impact, beyond the accumulation of biological knowledge, is an increase in our capacity to diagnose disease. Therapeutic benefits follow, but significantly more slowly. Herein lies a potentially frustrating paradox. When biomedical knowledge is undergoing rapid growth (as it is at present) we can anticipate a widening of the gap between our capacity to diagnose and our capacity to treat disease. For this reason it is fundamentally important that increased effort be focused on realising the therapeutic potential of advances in biological knowledge. Such an effort demands that we explore entirely new therapeutic paradigms and surmount the formidable translational challenges inherent in taking new therapies from the laboratory bench to the patient bedside. Gene Therapy, or "the use of genes as medicine" is one such paradigm, with immense but largely unrealised potential. The Gene Therapy Research Unit, a joint initiative of the The Children's Hospital at Westmead and Children's Medical Research Institute, was established in recognition of this potential and the need for a research environment that combines both basic science and clinical excellence. The research activities of the Unit reflect the challenge outlined above.

Main : Projects : Publications : Staff : Contact