Finding cures for children's genetic diseases


Gene Therapy Research Unit

The Gene Therapy Research Unit finds ways to correct genetic diseases in children. We are a joint initiative of Children’s Medical Research Institute and The Children’s Hospital at Westmead (CHW). Our primary goals are to develop more effective gene therapy methods and to translate basic research progress into improved health outcomes for children.
Clinical trials are underway to correct SCIDX1-deficiency (“boy in the bubble” disease) and to improve chemotherapy treatment of paediatric cancers. We are also currently working toward a new clinical trial to correct inherited liver disease (OTC and ASS deficiency urea cycle defects).

“Realising the therapeutic potential of progress in the laboratory is our ‘raison d’etre’, so we are particularly pleased to have three clinical trials under development or close to initiation. This is a major achievement and places our group at the forefront of this exciting field in Australia.” - Ian Alexander

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