Gene Therapy Research Unit

Our ultimate aim is to find treatments for genetic disease by using genes as medicine.  A joint initiative of Kids Research 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.

Contact us for advice and assistance on paediatric gene therapy clinical trials

The research team

Professor Ian Alexander, Gene Therapy Research Unit Head

As a Senior Staff Specialist and Clinical Geneticist at The Children’s Hospital at Westmead, Ian’s research works towards a day when he will be able to tell parents he not only knows the cause of their child’s disorder – but how to cure it. His training and day-to-day activities in clinical medicine and laboratory research reflect his interest in translating research progress into improved health outcomes for children.

Ian pioneered gene transfer technology in Australia – where repaired genes are transferred back to the body efficiently via vectors derived from viruses. His team became the first in Australia to treat a genetic disease (SCID-X1) by gene therapy. More information can be found on Ian's University of Sydney profile page.

> Other Research Team Members

  • Margot Latham, Administration and Program Manager, email:
  • Dr Sharon Cunningham, Research Fellow
  • Dr Samantha Ginn, Research Fellow
  • Dr Grant Logan, Research Fellow
  • Cindy Zhu, Senior Research Assistant
  • Emma Doroudian, Research Assistant
  • Neeta Khandekar, Research Assistant
  • Caitlin Lucas, Research Assistant
  • Eva van Dijk, Research Assistant
  • Sharntie Christina, PhD student
  • Dr Lara Graves, PhD student
  • James Kerr, PhD student
  • Mawj Mandwie, PhD student
  • Dr Kathryn Mullany, PhD student

Key publications

Smith RH*, Hallwirth CV*, Westerman M, Hetherington NA, Tseng Y, Cecchini S, Virag T, Ziegler M, Rogozin IB, Koonin EV, Agbandje-McKenna M, Kotin RM and Alexander IE (2016) Germline viral “fossils” guide in silico reconstruction of a mid-Cenozoic era marsupial adeno-associated virus. Scientific Reports. Online 5th July; 6:28965 DOI: 10.1038/srep28965

Hallwirth C, Garg G, Peters T, Kramer B, Malani NV, Hyman J, Ruan X, Ginn SL, Hetherington NA, Veeravalli L, Shahab A, Ranganathan S, Wei C, Liddle C, Thrasher A, Bushman FD, Buckley M and Alexander IE (2015) Coherence analysis discriminates between retroviral integration patterns in CD34+ cells transduced under differing clinical trial conditions. Molecular Therapy - Methods & Clinical Development eCollection Apr 29; 2:15015

Cunningham SC, Siew S, Hallwirth CV, Bolitho C, Sasaki N, Garg G, Michael IP, Hetherington NA, Carpenter K, de Alencastro G, Nagy A and Alexander IE (2015) Modeling Correction of Severe Urea Cycle Defects in the Growing Murine Liver using a Hybrid rAAV/piggyBac Transposase Gene Delivery System. Hepatology 62(2):417-428

Deyle DR, Hansen SR, Li LB, Burt AA, Cornea AM, Alexander IE, Stamatoyannopoulos J, Wei C-L and Russell DW (2014) A Genome-Wide Map of Human Gene Targeting. Nature Structural & Molecular Biology 21(11):969-975

Deakin CT, Alexander IE, Deakin JJ, Ginn SL, Young P, Humphries D, Suter CM and Hallwirth CV (2014) Impact of next generation sequencing error on the analysis of barcoded plasmid libraries of known complexity and sequence. Nucleic Acids Research 42(16):e129

Tay SS, McDonald D, Wood NA, Roediger B, Sierro F, McGuffog CM, Alexander IE, Bishop GA, Weninger W, McCaughan GW, Bertolino P and Bowen DG (2014) Antigen expression level threshold tunes the fate of CD8 T cells during primary hepatic immune responses. Proc. Natl. Acad. Sci. USA 111(25):E2540-E2549

Lisowski L, Dane AP, Chu K, Zhang Y, Cunningham SC, Wilson EM, Nygaard S, Grompe M, Alexander IE and Kay MA (2014) Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature 506(7488):382-386

Kojima Y, Kaufman-Francis K, Studdert JB, Steiner KA, Power MD, Loebel DAF, Jones V, Hor A, de Alencastro G, Logan G, Teber ET, Tam OH, Stutz MD, Alexander IE, Pickett HA and Tam PPL (2014) The transcriptional and functional properties of mouse epiblast stem cells resemble the anterior primitive streak. Cell Stem Cells 13:1-14

Kok CY, Cunningham SC, Carpenter KH, Dane AP, Siew SM, Logan GJ, Kuchel PW and Alexander IE (2013) Adeno-associated virus-mediated rescue of neonatal lethality in argininosuccinate synthetase deficient mice. Molecular Therapy 21(10):1823-1831

Deakin CT, Alexander IE, Hooker CA and Kerridge IH (2013)  Gene therapy researchers’ assessments and perceptions of risks in clinical trials. Molecular Therapy 21(4): 806-815