helenHelen Abud is a laboratory head and academic within the Cancer program of the Biomedicine Discovery Institute at Monash University.  Following her undergraduate degree at Melbourne University, she initially trained at the Walter and Eliza Hall Institute before undertaking her doctorate at Oxford University in cell and developmental biology. This was followed by postdoctoral training in the Department of Anatomy (Oxford), Peter MacCallum Cancer Centre (Melbourne) and the Ludwig Institute for Cancer Research (Melbourne). A/Prof Abud’s current research is centred on understanding the molecular mechanisms and environmental influences that regulate stem cells within normal tissues and tumours.  A/Prof Abud has a particular interest in molecules that promote intestinal epithelial development and regeneration following damage and how these factors may be altered in degenerative diseases and colon cancer.  She collaborates with clinicians at Cabrini Hospital to collect and grow organoids from normal colonic epithelium, primary and metastatic colorectal tumours.  These organoids are currently being used for a variety of biological studies including response to drug treatments and environment.



Christine Chio received her PhD from the University of Toronto under the mentorship of Dr Tak Wah Mak. During her graduate training, Dr. Chio studied the interplay between inflammation and cancer development. Specifically, she generated the genetic knockout for an adaptor molecule called TRADD, and demonstrated its essential role in both Toll like receptor and Tumor necrosis factor receptor signalling. Subsequently, Dr. Chio joined the lab of Dr. David Tuveson at Cold Spring Harbor Laboratory to pursue her postdoctoral training.  Her research endeavor in Dr Tuveson’s laboratory focused on understanding the role of cellular redox regulation, which is a critical biological dependency in any neoplasm and especially so in pancreatic cancer, being one of the most hypoxic solid tumors. While developing this project, she was involved in the establishment of a new organoid culture system for studying mouse and human pancreatic cancer progression. This model system offers unique advantages as it supports the growth of normal pancreatic ductal cells and each stage of the disease from both mouse and human specimens. Using this model system, she further identified that the protein synthesis machinery in pancreatic cancer cells is highly susceptible to changes in cellular redox, revealing a potential therapeutic strategy for this disease.



Peter Currie received his PhD in Drosophila genetics from Syracuse University, New York, USA.  He undertook postdoctoral training in zebrafish development at the Imperial Cancer Research Fund (now Cancer Research UK) in London, UK.  He has worked as an independent laboratory head at the UK Medical Research Council Human Genetics Unit in Edinburgh, UK and the Victor Chang Cardiac Research Institute in Sydney, Australia where he headed a research programme focused on skeletal muscle development and regeneration.

His work is centred on understanding how the small freshwater zebrafish is able to build and regenerate skeletal muscle.

In 2016 he was appointed Director of the Australian Regenerative Medicine Institute at Monash University in Melbourne, Australia.  He is a recipient of a European Molecular Biology Organization Young Investigators Award and a Wellcome Trust International Research Fellowship and currently is a Principal Research Fellow with the National Health and Medical Research Council in Australia.

Prof Currie, along with Dr Georgina Hollway, from the Garvan Institute of Medical Research, and Dr Phong Nguyen of the Australian Regenerative Medicine Institute at Monash University, won the UNSW 2015 Eureka Prize for Scientific Research. They were awarded the prize in recognition of their groundbreaking research into stem cell generation.



Partha Das – I did my BSc from University of Calcutta, India and Masters from University of Pune, India. After finishing my MSc, I was selected for another Master program in Molecular Biology at International Max Planck Research School, Gottingen, Germany. Then, I moved to Gurdon Institute, University of Cambridge, UK to do my PhD with Eric Miska to study- Role of short RNAs/ piRNAs in Caenorhabditis elegans germline development. For my post-doctoral research, I joined Dr. Stuart Orkin’s laboratory at Children’s Hospital Boston/DFCI, Harvard Medical School to study how chromatin changes regulate transcriptional gene regulation in embryonic stem cells (ESCs). My present and future studies elucidate how transcription factors (TFs) and epigenetic regulators, along with small RNAs and lncRNAs regulate gene expression in ESCs and differentiated cells under normal and pathological conditions.



Uta Griesenbach is a Professor in Molecular Medicine at the National Heart and Lung Institute, Imperial College London. She joined the Department of Gene Therapy at Imperial College in 1997 having earlier graduated from the University of Bonn (Germany) and completed a PhD at the Hospital for Sick Children, Toronto Canada and the University of Bonn.

Uta is a Strategy Group Member of the UK Cystic Fibrosis Gene Therapy Consortium (, a member of the Chronic Supporative Lung Disease (CSLD) Consortium, President (from April 2017) of the British Society for Gene and Cell Therapy ( and is the Gene Therapy advisor on a National Research Ethics Comittee.

Uta‘s reserach interests are related to the development of novel gene therapy-based treatments for cystic fibrosis and other inflammatory lung diseases as well as haemophilia and include vector and biomarker development, toxicology and GMP-vector production. Uta is Co-Investigator on several gene therapy trials, including a recently completed non-viral Phase IIb study. In addition, Uta is interested in developing the next generation of gene and cell therapists and in ths role is Programme Director of the MSc in Genes, Drugs and Stem cells and lead of the Genome-based therapeutics module of the MSc in Human Genomics at Imperial College.


Julian Heng attained his Bachelor’s of Science from UWA (1993-1996) followed by a PhD in developmental neuroscience at the University of Melbourne (1998-2002), working at the Howard Florey Institute.  He then trained as a CJ Martin Fellow and then as an MRC Career Development Fellow at the National Institute for Medical Research (UK) from 2004 to 2008. In 2010, Julian was appointed Group Leader at the Australian Regenerative Medicine Institute (Clayton, Australia) and received a Career Development Award (Level 1, NH&MRC). He then relocated his research to the Harry Perkins Institute of Medical Research (Perth, Western Australia) to establish the Brain Growth and Disease Laboratory.

Julian investigates the genetic basis for brain development and neurological disease. In the last five years, he has discovered 6 novel causative genes for structural brain malformations in children. To translate his gene discovery work, he collaborates with PathWest (a molecular diagnostics facility in Perth, WA) to implement a novel gene chip assay which screens for causative mutations in children diagnosed with a brain disorder. His research has been cited more than 1550 times (>960 times since 2012) (Source: Google Scholar).


Kimberly Homan joins us from Harvard University where she is currently a Research Associate at the Wyss Institute for Biologically Inspired Engineering. Kimberly started her scientific career at the University of Arizona where she earned a chemical engineering degree. She then took a break from science and was commissioned an officer in the United States Marine Corps where she served as a logistics officer for 6 years. After her service, she attended the University of Texas at Austin where she earned a Ph.D. in biomedical engineering. While in Austin, Texas she started a company based on the biomedical imaging contrast agents she developed in her graduate work. Her company, NanoHybrids Inc, is still based in Austin, and has brought several lines of unique gold nanoparticles to market. Kimberly now applies her biomedical engineering expertise to tissue engineering, and has worked for the last 4 years to use 3D printing to create functional living tissue. In the laboratory of Jennifer Lewis at Harvard University and co-advised by Annie Moisan at Roche, she has built kidney tissue on perfusable chips that can be used for drug screening, mechanistic safety, and disease modeling.



Sara Howden has international experience in the area of genetic editing in human pluripotent stem cells. A recipient of an NHMRC C.J. Martin Overseas Fellowship, she trained in the laboratory of Prof. Jamie Thomson, a pioneer in human pluripotent stem cell biology and in the reprogramming of somatic cells to induced pluripotent stem cells. In 2011 she was the primary investigator on a study that was first to describe targeted gene repair of patient-specific iPS cells. Having returned to Australia, Dr. Howden is now an emerging leader in the generation and editing of hPSCs and has developed novel protocols for the efficient generation of new lines using CRISPR/Cas9.




Brian Kaspar‘s research focuses on basic and translational studies related to neurological and neuromuscular disorders. The laboratory has strengths in animal models of neurodegenerative and neuromuscular disease, gene delivery, and stem cell biology. A main focus of the Kaspar laboratory is centered on the mechanism(s) of neurodegeneration in Amyotrophic Lateral Sclerosis (ALS) and Spinal Muscular Atrophy (SMA). We employ rodent models of this disease to investigate various cell type involvements in disease onset and progression. Furthermore, we are actively developing novel methods to deliver genes and therapies more efficiently to the nervous system and testing novel targets to combat this debilitating, lethal disease. The laboratory also investigates the biological control of embryonic and adult derived stem cells. Our current studies with stem cells are evaluating cell cycle regulation along with developing methods for intricate control of differentiation to defined cellular phenotypes, such as complex motor neurons. Finally, our laboratory works on muscle enhancing strategies in order to combat musculoskeletal disorders. We have identified that follistatin is a potent antagonist of myostatin, and when delivered by a one-time gene delivery to skeletal muscle, enhances muscle size and mass. Interestingly, our work has demonstrated that follistatin reduces the inflammatory environment in dystrophic muscles and induces improvements in functional strength in rodent models of Duchene Muscular Dystrophy. We are currently in a Phase I/II human clinical trial for Becker Muscular Dystrophy and Inclusion Body Myositis using our expertise in Adeno Associated Virus.  Furthermore, we have advanced human trials for gene delivery in Spinal Muscular Atrophy.   We utilize a variety of viral vectors including Adenovirus, Adeno Associated Virus, Retrovirus and Lentivirus to answer important questions


eddyEddy Kizana is a Cardiologist at Westmead Hospital and Senior Lecturer, Sydney Medical School. He is Group Leader and Faculty at WIMR. He was awarded his MBBS (Hons) (1995) and PhD (2006) from The University of Sydney. He completed his cardiology training before embarking on an NHF-funded PhD in gene transfer technology with Ian Alexander at the CMRI. He undertook post-doctoral research training at the Johns Hopkins University with Eduardo Marbán and was awarded the Michel Mirowski, MD Fellowship from the Heart Rhythm Society. In addition to developing gene therapy strategies targeting cardiac arrhythmias, he was also collaboratively involved with molecular and stem cell-based therapies for heart failure and hypertrophy. To dovetail his clinical and research interests, he has established a research group at WIMR whose focus is to develop novel gene therapy approaches for common cardiac arrhythmias. Through local and international collaborative efforts his group has developed capabilities for producing viral vectors for gene transfer in animal models of conduction and arrhythmias.


leszekLeszek Lisowski received his PhD from Cornell University Weill Graduate School of Medical Sciences in New York and performed his research in the lab of Dr Michel Sadelain at the Memorial Sloan Kettering Cancer Center. Here he developed improved lentiviral vectors for the treatment of beta-thalassemia and hemophilia and performed extensive work on the assessment of vector toxicity and the risk of insertional oncogenesis. Subsequently he did his postdoctoral training at Stanford University School of Medicine, under the supervision of Dr Mark A. Kay, focusing on the biology of adeno-associated virus (AAV) and vector-mediated gene transfer. After completing his postdoctoral training, Dr Lisowski was recruited by the Salk Institute for Biological Studies in La Jolla, CA to head the Gene Transfer, Targeting and Therapeutics (GT3) Facility, where he expanded his expertise in viral vectors by working with Rabies virus, AAV, adenovirus and Vesicular stomatitis virus. He was then recruited by the Children’s Medical Research Institute at Westmead, to establish a Vector and Genome Engineering Facility and an independent Translational Vectorology Research Group (TVG). TVG focuses on the development of new AAV selection technologies, AAV-based gene editing technology as well as understanding fundamental AAV biology related to the intimate evolutionary relation between AAV and human liver. He also holds a conjoint appointment of Senior Lecturer at the University of Sydney and Visiting Associate Professor in the Department of Genetic Engineering at the Military Institute of Hygiene and Epidemiology (WIHE) in Poland.


amitAmit Nathwani, a NIHR Senior Investigator, is the Director of the Katharine Dormandy Haemophilia Centre at the Royal Free Hospital and Professor of Haematology at UCL. He is internationally recognised for his pioneering work in gene therapy and is the recipient of the ESGCT Outstanding Achievement Award and the Human Gene Therapy Award amongst others. Prof Nathwani developed a novel gene transfer approach that enabled the first successful trial of gene therapy in a bleeding disorder- haemophilia B and this has now become the platform for a variety of genetic disorders. He has also developed a novel approach for gene therapy of haemophilia A that has resulted in normalisation of coagulation factor VIII levels and correction of bleeding diathesis in severely affected patients. His group is working on other translational gene transfer approaches for a range of monogenetic disorders as well as malignancies.




Elizabeth Ng‘s interests lie in the regulation of human pluripotent stem cell differentiation to blood and endothelium, with the aim of generating definitive hematopoietic lineages, including hematopoietic stem cells, for transplantation and disease modeling. Her work is facilitated by the use of human pluripotent stem cell lines in which fluorescent reporter genes identify cells forming endothelium and hematopoietic cells. She has recently developed a protocol that enables the differentiation of hPSCs to haemogenic vasculature similar to the human aorta-gonad-mesonephros, the site at which the first haematopoietic stem cells emerge during embryonic development.


susieSusie Nilsson received her Ph.D. in Pathology from Melbourne University before completing post-doctoral studies at the University of Massachusetts. On his return to Australia, Susie took up a position at the Peter MacCallum Cancer Institute where she became a laboratory head in 2002. In 2005 she moved to the Australian Stem Cell Centre prior to being appointed as an ARC Future Fellow and a Group Leader at CSIRO in 2009. She became a group leader at ARMI, Monash University in 2013 and was appointed as a CSIRO OCE Science Leader in 2014. Currently she has an adjunct Professor appointment at Monash University.  The specific goal of her laboratory is to understand hemopoiesis and therefore begin to address the changes and their effects in hemopoietic disease. She has focused much of her independent research career establishing models to identify where HSC reside in the BM, the cellular and extracellular components in that microenvironment as well as the mechanisms through which these regulate HSC fate. She currently serves as Associate editor for the International Journal of Hematology and Experimental Hematology.



Michael O’Connor uses human pluripotent cells to model eye diseases including cataract. He received his PhD from the University of Sydney in 2005, for regenerating and characterising functional rat lenses in vitro. During postdoctoral studies in Vancouver, Canada, he identified genes related to pluripotency, and co-led commercial development of the now gold-standard pluripotent stem cell medium mTeSR1 and TeSR2 with Stem Cell Technologies. During his tenure as President of the Australasian Society for Stem Cell Research he led a variety of public- and policy-related projects aimed at advancing the development of a responsible and effective stem cell industry in Australia.


clareClare Parish heads the Stem Cells and Neural Development laboratory at the Florey Institute of Neuroscience & Mental Health. She obtained her PhD from Monash University, Australia in 2002 and subsequently spent 5 years of postdoctoral training at the Karolinska Institute (Stockholm, Sweden). She returned to Australia in 2007 to establish her own research group.  She has been the recipient of numerous fellowships including the Viertel Senior Medical Research Fellowship, NHMRC, Australia CDF and Human Frontiers Science Program fellowship as well as both national and international grant funding. She has >60 peer review publications and several patents related to her research focused on the development of stem cell-based therapies for brain repair. Her primary focus is on cell transplantation and the use of bioengineered scaffolds for the treatment of Parkinson’s disease and stroke.


stevenSteven Petrou is a Professor of Translational Neuroscience in the Department of Medicine at the University of Melbourne and Deputy Director of the Florey Institute for Neuroscience and Mental Health.  His research focuses on the disease biology of epilepsy and recent focus has been on unravelling the genetic, biophysical and neurophysiological consequences of de novo mutations in the epileptic encephalopathies.  He leads a large team of interdisciplinary researchers that can address mutant analysis at multiple structural and functional levels needed for a complete understanding of the emergent pathomechanisms.  His group also works toward the generation of disease mechanism targeted therapies and recent highlights include disease mechanism targeted strategies for Dravet Syndrome and Epilepsy of Infancy with Migrating Focal Seizures.  He works closely with commercial collaborators and has founded two US based biotech companies, Praxis Precision Medicines and RogCon in the USA.


enzoEnzo Porrello received his PhD in Physiology from The University of Melbourne in 2009. He was subsequently awarded an NHMRC/NHF C.J. Martin fellowship to undertake postdoctoral training at UT Southwestern Medical Center in Dallas, USA, in the laboratory of Prof. Eric Olson. Dr Porrello returned to Australia in 2012 to establish the Cardiac Regeneration Laboratory in the School of Biomedical Sciences at The University of Queensland. He was subsequently recruited to the Murdoch Childrens Research Institute and The University of Melbourne where he will head the Cardiac Regeneration Laboratory from mid-2017. Dr Porrello currently holds a joint NHMRC Career Development Fellowship and Heart Foundation Future Leader Fellowship. His research focuses on understanding the transcriptional mechanisms that control cardiac regenerative capacity during neonatal life. Dr Porrello’s research has been recognized by a number of awards including the NHF Paul Korner Innovation Award, Heart Foundation Researcher of the Year, UQ Foundation for Research Excellence Award, A.K. McIntyre Prize (Australian Physiological Society) and the UT Southwestern Postdoctoral Achievement Award


elizabeth pElizabeth P. Rakoczy (PhD) is the foundation Professor of Molecular Ophthalmology at the Lions Eye Institute, The University of Western Australia. Prof Rakoczy’s research interest focuses on Age Related Macular Degeneration (AMD), diabetic retinopathy and retinal degenerations. Her laboratory developed several animal models for eye diseases, including the ‘Akimba’ mouse model for advanced diabetic retinopathy that has been licensed around the world. She pioneered the concept of mutation-independent gene therapy called secretion gene therapy or ‘biofactory’ for the treatment of wet age related macular degeneration. The technology was licensed by Avalanche Biotechnologies CA, USA in 2010. She has published more than 180 scientific papers and several patents and is a regular reviewer for The Lancet, Human Gene Therapy, Gene Therapy, NHMRC and the Wellcome Trust.




Alexandra Sharland is a medical graduate with a longstanding interest in the mechanisms of transplantation tolerance and rejection, and in the use of gene transfer to promote the development of graft-specific tolerance and to limit the graft injury and inflammation which oppose tolerance induction. She is currently Associate Professor in Transplantation Immunobiology at the University of Sydney, and leads a research group based in the Charles Perkins Centre.




Nicholas Smith is a neurologist specialising in the management of paediatric and adult neurometabolic diseases, with a particular interest in the disorder of lysosomal function.

His clinical and scientific research interests focus upon defining the pathological mechanisms underpinning neurological forms of lysosomal disease to inform the development of novel therapeutic approaches, including gene transfer therapies, for children and adults with lysosomal and related neurodegenerative disorders.

masatoshiMasatoshi Tagawa graduated with MD from the School of Medicine, Chiba University (Chiba, Japan) and completed the graduated course in Chiba University (awarded PhD) in 1984. He spent a few years in Stanford University (CA, USA) as a postdoctoral fellow and became an assistant professor at Chiba University. He then moved to Chiba Cancer Center Research Institute (Chiba, Japan) as the Head in Division of Pathology and Cell Therapy, and became a professor of Graduate School of Medicine at Chiba University. He is also a visiting professor of Hebei Medical University and Shenyang Pharmaceutical University, China. He is currently a council member of the Japan Society of Gene Therapy, president of the International Society of Cell and Gene Therapy of Cancer, and vice director for Incorporated Non-profit Organization Asian Pacific Consortium of Gene and Cell Therapy. He also serves as an editor of Cancer Gene Therapy and other journals. His primary research field is immunology, molecular biology and oncology, and he had been investigating cytokine-mediated immune responses and suicide gene therapy. He is currently working on mesothelioma at preclinical and clinical research levels.


lukLuk H. Vandenberghe is Assistant Professor at Harvard Medical School and directs the Grousbeck Gene Therapy Center at the Schepens Eye Research Institute and Mass Eye and Ear in Boston, Massachusetts, USA. Dr. Vandenberghe trained as a Biochemical Engineer at the University of Leuven, Belgium after which he did his doctoral work in Biomedical Sciences on ‘The structural basis for adeno-associated virus (AAV) as a gene therapeutic’.  His work throughout the years has focused on HIV, Adenovirus, and AAV in the laboratories of Drs. Debyser (University of Leuven, Belgium), June, Wilson, and Bennett (University of Pennsylvania, USA). In 2012, Dr. Vandenberghe established his independent research group that (i) translates – primarily retinal – gene therapy programs, (ii) develops enabling gene transfer technologies, and (iii) studies host-vector interactions relevant to gene therapy applications. Dr. Vandenberghe has produced over 50 peer reviewed publications and 20 patents to technologies, several of which are licensed and being evaluated in the clinic. He is also a scientific co-founder of GenSight Biologics, a retinal gene


profesor christineProfessor Christine Wells is the founding Director of the University of Melbourne Centre for Stem Cell Systems. She is the deputy program leader for Stem Cells Australia, an ARC funded $21M special research initiative to bring together leading Australian stem cell scientists.  Christine is a genome biologist interested in tissue injury and repair. She leads a program of research in biological data integration and visualization for the stem cell community, including method development leading to gene discovery and characterization of stem cell subsets and innate immune cells.

Professor Wells has published over 92 scientific journal articles, in the leading scientific literature, including landmark studies in mapping gene architecture and function. She has developed several open source software programs, including the stem cell collaboration resource, which has a global audience, and which hosts the largest compendium of curated stem cell data. This resource is used to generate definitive molecular signatures of stem cell subsets and their differentiated progeny, including accurate classification of MSC from different tissues. Over the last 16 years, Christine has worked with the Japanese genome consortium, FANTOM, led from the RIKEN Center for Life Science Technologies (CLST), Yokohama, on discovery of the genetic toolkit used by cells and tissues during development, growth, aging and disease.



Steve Wilton has developed a novel therapy for Duchenne muscular dystrophy using “gene patches” to restore functional dystrophin expression, with Exondys 51, a morpholino oligomer being granted accelerated approval by the US Food and Drug Administration in September 2016.  This work has been recognized as Western Australian Innovator of the Year (2012), Australia Museum Eureka Award for Translational Research (2013), an ASBMB LabGear Discovery award (2014) and he was a finalist in Western Australian of Year (2016). He is the Director of the Perron Institute for Neurological and Translational Science and holds the Foundation Chair in Molecular Therapies at Murdoch University.


Plenary: Clinical translation of cell and gene therapy
Professor Amit Nathwani



Royal Free Hospital, University College London, UK

Broad topic: Advances in AAV gene therapy to treat haemophilia B




Plenary: Disease modeling and drug screening
Dr Christine Chio



Cold Spring Harbor Laboratory, Cold Spring Harbor, USA

Broad topic: Developing effective therapies using organoids of pancreatic ductal cancer




Plenary: Regenerative Medicine and Bioengineering
Dr Kimberly Homan



Wyss Institute, Harvard, USA

Broad topic: Can 3D printing be used to create organs?




AGCTS focused session – Vector development 
A/Professor Luk Vandenburghe

Schepens Eye Research Institute and Massachusetts Eye and Ear Infirmary (both affiliates of Harvard Medical School)

Title: Unlocking therapeutic gene transfer for broad application: progress and challenges





More speakers added soon – watch this space!