Gene Therapy for Genetic Disease: Pushing the Technological Boundaries

When:  Nov 29, 2021 from 09:00 to 10:00 (AEST)
Sunday, November 28 - 15:00 PST / 18:00 EST
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Gene Therapy for Genetic Disease: Pushing the Technological Boundaries

Lecture 2 of the ISCT ANZ Cell & Gene Therapy Translation Lecture Series
Organized by the ISCT Australia & New Zealand Regional Executive Committee

Lecture Description:

The ISCT ANZ Cell & Gene Therapy Translation Lecture Series aims to provide ISCT members innovative and ground-breaking research in cell and gene therapy. These lectures will be presented by world-leading experts and will focus on cutting edge advances in cell and gene therapy development, manufacturing, and clinical application.

In the second lecture of the series, Professor Ian Alexander will address the following topics:

- Overview of the history and current status of the gene therapy field
- The dependence of progress on advances in gene transfer technology
- Which gene transfer technologies are succeeding in the clinic and why? 
- Lessons learnt and emerging challenges
- The exciting possibilities ahead

Co-Chaired By: 

Rajiv Khanna, PhD AO
ISCT ANZ Regional Vice-President
QIMR Berghofer Medical Research Institute

Professor Rajiv Khanna obtained his doctorate degree from India and undertook his post-doctoral training at the Queensland Institute of Medical Research (QIMR). He is the Coordinator of QIMR Berghofer Centre for Immunotherapy and Vaccine Development. He holds Senior Principal Research Fellowship from the National Health and Medical Research Council of Australia and was also appointed as Honorary Professor at the University of Queensland and Griffith University, Brisbane. Professor Khanna is a Fellow of Australian Academy of Health and Medical Sciences.


Madison Paton, BSc (Hons), PhD
ISCT ESP Committee Member
Cerebral Palsy Alliance

Dr Paton is a Research Fellow at the Cerebral Palsy Alliance Research Institute and is affiliated with University of Sydney and Monash University. She received her PhD in 2018 investigating stem cell therapies to protect the developing brain. She has been working in the field of cell therapies and cerebral palsy for more than 8 years and has transitioned from working in the lab, to helping develop clinical trials that drive the Australian research pipeline. In her current role, Dr Paton works on projects that apply early cell therapy treatment for neuroprotection and is particularly interested in utilising umbilical cord blood and mesenchymal stem cell therapies to transform clinical practice.


Ian Alexander, BMedSci, MBBS (Hons), PhD, FRACP (paeds), HGSACG, FAHMS
University of Sydney

Professor Alexander is physician scientist with paediatric sub-specialty training in genetic/metabolic disease and over 25 years research experience in the gene therapy field. He heads the Gene Therapy Research Unit within the Children’s Medical Research Institute and Sydney Children’s Hospitals Network where he also holds appointments as a senior staff specialist and Director of laboratory research. Since undertaking post-doctoral studies in Seattle in the laboratory of A. Dusty Miller, he has pursued a primary interest in virus mediated gene transfer to the paediatric liver with a strong translational focus on urea cycle defects, most notably OTC deficiency. He is recognized internationally for significant basic and applied contributions to the science of gene transfer, experimental therapeutics and early phase human clinical trials, and in 2015 was appointed a Fellow of the Australian Academy of Health and Medical Science in recognition of contributions to the establishment of the gene therapy field in Australia.