JCSMR School Seminar Series - Liver-targeted gene therapy: a 50 million year odyssey

Professor Ian Alexander, Head - Gene Therapy Research Unit, a joint initiative of Children’s Medical Research Institute and The Children’s Hospital at Westmead (CHW), Sydney, NSW

Genetic manipulation of the human liver holds immense therapeutic promise. This presentation will explore the challenge of fulfilling this promise from a gene delivery perspective, taking into account the unique biology of the liver and demands imposed by specific disease targets. Special attention will be given to evolving technologies based on recombinant Adeno-Associated Virus (rAAV), with insights from paleovirology and speculation about the exciting near future. 

Professor Alexander is head of the Gene Therapy Research Unit, a joint initiative of Sydney Children’s Hospitals Network and Children’s Medical Research Institute in Sydney. Within the hospital he also holds appointments as a senior staff specialist and Director of laboratory research. His training and day-to-day activities in both clinical medicine and laboratory research reflect his interest in translating research progress into improved health outcomes for children. After completing specialty training in paediatrics at Prince of Wales Children’s Hospital he obtained a PhD in Molecular Biology from the Garvan Institute in Sydney before completing clinical genetics training at the Murdoch Institute in Melbourne. He then undertook postdoctoral studies at the Fred Hutchinson Cancer Research Institute in Seattle before returning to Australia to take up his current position. He has since established a translational research program and developed the specialised infrastructure and skill sets required to take promising novel therapies through to clinical application. His team became the first in Australia to treat a genetic disease (SCID-X1) by gene therapy and are recognised leaders in the establishment of this exciting field in Australia. His specific expertise and interests include virus-mediated gene transfer with a focus on metabolic liver disease and primary immunodeficiencies. Within Sydney Children’s Hospitals Network, which serves a third of the entire paediatric population of Australia, he is currently leading the development of a major research initiative in Genomics Rare Disease. He was the inaugural president of the Australasian Gene Therapy Society in 2001 and was Chair of the NHMRC Cellular Therapies Advisory Committee (2007-2009 triennium). He is currently Chair of the International Committee of the American Society of Gene and Cell Therapy, is the Australasian Regional Editor of Current Gene Therapy and Associate Editor for Human Gene Therapy and The Journal of Gene Medicine. In 2015 he was appointed as a Fellow of the Australian Academy of Health and Medical Sciences and a Visiting Professor at University College London.

Date & time

12–1pm 7 April 2017


The Finkel Lecture Theatre, The John Curtin School of Medical Research, Building 131 Garran Road, ANU


Host: Professor Simon Foote


 Ceri Flowers
 +61 2 6125 2589

Updated:  19 July 2018/Responsible Officer:  Director, JCSMR/Page Contact:  Web Manager