Gene Therapy Research Unit

Gene Therapy Research Unit

This page is a research page on Kids Research. Also see the Children’s Medical Research Institute’s (CMRI) website.

CMRI page

The Gene Therapy Research Unit is a joint initiative of Children’s Medical Research Institute and Kids Research based at The Children’s Hospital at Westmead. To treat genetic disorders, we either repair the body’s faulty genes or replace them with functioning genes using vector technology. Our particular interests are genetic disorders of the liver and the body’s immune response to gene therapeutics.


Our main objective is to enable better health outcomes for children by facilitation access to world class gene therapeutics.

We achieve this through exploring ways of translating new therapies, developed both in our laboratory and internationally, into successful treatments for children. 


  • We are Australia’s leading group specialising in gene transfer and gene editing technology and the first in Australia to treat a child with gene therapy.
  • Our translational research program has defined a clear path for clinical translation of gene therapeutics. We provide the specialised infrastructure and skill sets required to take promising novel therapies through to clinical application. 
  • We have provided valuable insights into host-vector responses from our work with the University of Florida, in isolating the single largest collection of man-made human antibodies (or anti-Adeno-Associated Virus monoclonal antibodies).


Prof Ian Alexander

Prof Ian Alexander

Email Send email
Head, Gene Therapy Research Unit. Professor of Paediatrics and Molecular Medicine, Faculty of Medicine and Health, University of Sydney.
Related Links
Last updated Monday 19th February 2024