Gene Therapy Research Unit

Our ultimate aim is to find treatments for genetic disease by using genes as medicine.  A joint initiative of The Children's Hospital at Westmead and Children's Medical Research Institute, we focus on realising the therapeutic potential of rapid advances in biomedical knowledge.

To treat genetic disorders, we repair faulty genes and deliver them back into the body using vector technology. We are Australia’s leading group specialising in gene transfer technology and the first in Australia to treat a child with gene therapy.

Our particular interest is genetic disorders of the bone marrow and liver.  Our staff explore ways of translating new therapies developed in the laboratory into successful treatments for patients.