Professor Ian Alexander

Professor in Paediatrics & Molecular Medicine
Genetic Medicine, Children's Hospital, Westmead

C29 - Children's Hospital Westmead
The University of Sydney
NSW 2006 Australia

T: +61 2 9845 3071
F: +61 2 9845 1317
E: Send message

Research interests

Dr Alexander's specific expertise and interests include virus-mediated gene transfer with a focus on target organs including the liver and bone marrow, both of which have immense promise as target organs for the treatment of genetic disease in children. 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.

Current national competitive grants*


Cellular therapy for genetic liver disease exploiting induced pluripotent stem cells and liver progenitor cells
Alexander I, Tam P, Russell D, Yeoh G
NHMRC Project Grants ($770,175 over 3 years)

Analysis and manipulation of the genome-wide integration signatures of gamma-retroviral and lentiviral vectors in human haematopoietic stem cells
Alexander I, Ranganathan S, Cavazzana-Calvo M
NHMRC Project Grants ($591,125 over 3 years)

* Grants administered through the University of Sydney


2014 | 2013 | 2012 | 2011 | 2010 | 2009 | 2008


  • Lisowski, L., Dane, A., Chu, K., Zhang, Y., Cunningham, S., Wilson, E., Nygaard, S., Grompe, M., Alexander, I., Kay, M. (2014), Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature. 506(7488), 382-386. [Abstract]
  • Kojima, Y., Kaufman-Francis, K., Studdert, J., Steiner, K., Power, M., Loebel, D., Jones, V., Hor, A., de Alencastro, G., Logan, G., Teber, E., Tam, O., Stutz, M., Alexander, I., Pickett, H., Tam, P. (2014), The transcriptional and functional properties of mouse epiblast stem cells resemble the anterior primitive streak. Cell Stem Cell. 14(1), 107-120. [Abstract]


  • Cunningham, S., Kok, C., Spinoulas, A., Carpenter, K., Alexander, I. (2013), AAV-encoded OTC activity persisting to adulthood following delivery to newborn spf(ash) mice is insufficient to prevent shRNA-induced hyperammonaemia. Gene Therapy. 20(12), 1184-1187. [Abstract]
  • Dane, A., Wowro, S., Cunningham, S., Alexander, I. (2013), Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes. Gene Therapy. 20(4), 460-464. [Abstract]
  • Kok, C., Cunningham, S., Carpenter, K., Dane, A., Siew, S., Logan, G., Kuchel, P., Alexander, I. (2013), Adeno-associated Virus-mediated Rescue of Neonatal Lethality in Argininosuccinate Synthetase-deficient Mice. Molecular Therapy. 21(10), 1823-1831. [Abstract]
  • Gerace, D., Ren, B., Hawthorne, W., Byrne, M., Phillips, P., O'Brien, B., Nassif, N., Alexander, I., Simpson, A. (2013), Pancreatic transdifferentiation in porcine liver following lentiviral delivery of human furin-cleavable insulin. Transplantation Proceedings. 45(5), 1869-1874. [Abstract]
  • Deakin, C., Alexander, I., Hooker, C., Kerridge, I. (2013), Gene therapy researchers' assessments of risks and perceptions of risk acceptability in clinical trials. Molecular Therapy. 21(4), 806-815. [Abstract]
  • Ginn, S., Alexander, I., Edelstein, M., Abedi, M., Wixon, J. (2013), Gene therapy clinical trials worldwide to 2012 - an update. The Journal of Gene Medicine. 15(2), 65-77. [Abstract]
  • Cunningham, E., Tay, S., Wang, C., Rtshiladze, M., Wang, Z., McGuffog, C., Cubitt, J., McCaughan, G., Alexander, I., Bertolino, P., Sharland, A., Bowen, D., Bishop, G. (2013), Gene therapy for tolerance: high-level expression of donor major histocompatibility complex in the liver overcomes naive and memory alloresponses to skin grafts. Transplantation. 95(1), 70-77. [Abstract]


  • Logan, G., Alexander, I. (2012), Adeno-associated virus vectors: immunobiology and potential use for immune modulation. Current Gene Therapy. 12(4), 333-343. [Abstract]
  • Alexander, I., Kok, C., Dane, A., Cunningham, S. (2012), Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders. Journal of Inherited Metabolic Disease. 35(4), 641-645. [Abstract]
  • Michot, C., Hubert, L., Romero, N., Gouda, A., Mamoune, A., Mathew, S., Kirk, E., Viollet, L., Rahman, S., Bekri, S., Peters, H., McGill, J., Glamuzina, E., Farrar, M., von der Hagen, M., Alexander, I., Kirmse, B., Barth, M., Laforet, P., Benlian, P., Munnich, A., Jeanpierre, M., Elpeleg, O., Pines, O., Delahodde, A., de Keyzer, Y., de Lonlay, P. (2012), Study of LPIN1, LPIN2 and LPIN3 in rhabdomyolysis and exercise-induced myalgia. Journal of Inherited Metabolic Disease. 35(6), 1119-1128. [Abstract]
  • Chung, H., Lin, R., Logan, G., Alexander, I., Sachdev, P., Sidhu, K. (2012), Human induced pluripotent stem cells derived under feeder-free conditions display unique cell cycle and DNA replication gene profiles. Stem Cells and Development. 21(2), 206-216. [Abstract]
  • Ginn, S., Alexander, I. (2012), Gene therapy: Progress in childhood disease. Journal of Paediatrics and Child Health. 48(6), 466-471. [Abstract]
  • De Greef, E., Christodoulou, J., Alexander, I., Shun, A., O'Loughlin, E., Thorburn, D., Jermyn, V., Stormon, M. (2012), Mitochondrial Respiratory Chain Hepatopathies: Role of Liver Transplantation. A Case Series of Five Patients. In: JIMD Reports - Case and Research Reports, 2012/1. (pp.5-11).United Kingdom: Springer.


  • Both, G., Alexander, I., Fletcher, S., Nicolson, T., Rasko, J., Wilton, S., Symonds, G. (2011), Gene therapy: therapeutic applications and relevance to pathology. Pathology. 43(6), 642-56. [Abstract]
  • El-Hoss, J., Sullivan, K., Cheng, T., Yu, N., Bobyn, J., Peacock, L., Mikulec, K., Baldock, P., Alexander, I., Schindeler, A., Little, D. (2011), A murine model of neurofibromatosis type 1 tibial pseudarthrosis featuring proliferative fibrous tissue and osteoclast-like cells. Journal of Bone and Mineral Research. 27(1), 68-78. [Abstract]
  • Cunningham, S., Kok, C., Dane, A., Carpenter, K., Kizana, E., Kuchel, P., Alexander, I. (2011), Induction and Prevention of Severe Hyperammonemia in the spf(ash) Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery. Molecular Therapy. 19(5), 854-9. [Abstract]


  • Deakin, C., Alexander, I., Kerridge, I. (2010), The ethics of gene therapy: balancing the risks. Current Opinion in Molecular Therapeutics. 12(5), 578-585. [Abstract]
  • Sharland, A., Logan, G., Bishop, A., Alexander, I. (2010), Liver-directed gene expression using recombinant AAV 2/8 vectors -- a tolerogenic strategy for gene delivery?. Discovery Medicine. 9(49), 519-527. [Abstract]
  • Ginn, S., Liao, S., Dane, A., Hu, M., Hyman, J., Finnie, J., Zheng, M., Cavazzana-Calvo, M., Alexander, S., Thrasher, A., Alexander, I. (2010), Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and gammac Overexpression. Molecular therapy : the journal of the American Society of Gene Therapy. 18(5), 965-76. [Abstract]
  • Logan, G., Wang, L., Zheng, M., Coppel, R., Alexander, I. (2010), Antigen fusion with C3d(3) augments or inhibits humoral immunity to AAV genetic vaccines in a transgene-dependent manner. Immunology and cell biology. 88(2), 228-32. [Abstract]


  • Deakin, C., Alexander, I., Kerridge, I. (2009), Accepting risk in clinical research: is the gene therapy field becoming too risk-averse?. Molecular Therapy. 17(11), 1842-1848. [Abstract]
  • Dane, A., Cunningham, S., Graf, N., Alexander, I. (2009), Sexually Dimorphic Patterns of Episomal rAAV Genome Persistence in the Adult Mouse Liver and Correlation With Hepatocellular Proliferation. Molecular Therapy. 17(9), 1548-1554. [Abstract]
  • Cunningham, S., Spinoulas, A., Carpenter, K., Wilcken, B., Kuchel, P., Alexander, I. (2009), AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spf(ash) Mice. Molecular Therapy. 17(8), 1340-1346. [Abstract]
  • Laurence, J., Allen, R., McCaughan, G., Logan, G., Alexander, I., Bishop, G., Sharland, A. (2009), Gene therapy in transplantation. Transplantation Reviews. 23(3), 159-170. [Abstract]
  • Ginn, S., Cunningham, S., Zheng, M., Spinoulas, A., Carpenter, K., Alexander, I. (2009), In vivo assessment of mutations in OTC for dominant-negative effects following rAAV2/8-mediated gene delivery to the mouse liver. Gene Therapy. 16(6), 820-823. [Abstract]
  • Lee, A., Kahatapitiya, P., Kramer, B., Joya, J., Hook, J., Liu, R., Schevzov, G., Alexander, I., McCowage, G., Montarras, D., Gunning, P., Hardeman, E. (2009), Methylguanine DNA methyltransferase-mediated drug resistance-based selective enrichment and engraftment of transplanted stem cells in skeletal muscle. Stem Cells. 27(5), 1098-1108. [Abstract]
  • Laurence, J., Wang, C., Zheng, M., Cunningham, S., Earl, J., Tay, S., Allen, R., McCaughan, G., Alexander, I., Bishop, G., Sharland, A. (2009), Overexpression of indoleamine dioxygenase in rat liver allografts using a high-efficiency adeno-associated virus vector does not prevent acute rejection. Liver Transplantation. 15(2), 233-241. [Abstract]
  • Logan, G., Wang, L., Zheng, M., Ginn, S., Coppel, R., Alexander, I. (2009), Antigen-specific humoral tolerance or immune augmentation induced by intramuscular delivery of adeno-associated viruses encoding CTLA4-Ig-antigen fusion molecules. Gene therapy. 16(2), 200-10. [Abstract]
  • Liu, R., Ginn, S., Lek, M., North, K., Alexander, I., Little, D., Schindeler, A. (2009), Myoblast sensitivity and fibroblast insensitivity to osteogenic conversion by BMP-2 correlates with the expression of Bmpr-1a. BMC musculoskeletal disorders. 10(0), 51. [Abstract]


  • Lee, V., Wang, Y., Wang, Y., Zheng, D., Polhill, T., Cao, Q., Wu, H., Alexander, I., Alexander, S., Harris, D. (2008), Regulatory immune cells in kidney disease. American journal of physiology. Renal physiology. 295(2), F335-42. [Abstract]
  • Cunningham, S., Dane, A., Spinoulas, A., Logan, G., Alexander, I. (2008), Gene delivery to the juvenile mouse liver using AAV2/8 vectors. Molecular Therapy. 16(6), 1081-1088. [Abstract]
  • Alexander, I., Cunningham, S., Logan, G., Christodoulou, J. (2008), Potential of AAV vectors in the treatment of metabolic disease. Gene Therapy. 15(11), 831-839. [Abstract]
  • Curtin, J., Dane, A., Swanson, A., Alexander, I., Ginn, S. (2008), Bidirectional promoter interference between two widely used internal heterologous promoters in a late-generation lentiviral construct. Gene Therapy. 15(5), 384-390. [Abstract]
  • Baoutina, A., Alexander, I., Rasko, J., Emslie, K. (2008), Developing strategies for detection of gene doping. The journal of gene medicine. 10(1), 3-20. [Abstract]