Finding cures for children's genetic diseases


Gene Therapy Research Unit


Recent Publications

  1. The Transcriptional and Functional Properties of Mouse Epiblast Stem Cells Resemble the Anterior Primitive Streak. Kojima Y, Kaufman-Francis K, Studdert JB, Steiner KA, Power MD, Loebel DA, Jones V, Hor A, de Alencastro G, Logan GJ, Teber ET, Tam OH, Stutz MD, Alexander IE, Pickett HA, Tam PP. Cell Stem Cell. 2013 Oct 15. doi:pii: S1934-5909(13)00414-1. 10.1016/j.stem.2013.09.014. [Epub ahead of print]. PMID: 24139757.
  2. Adeno-associated virus-mediated rescue of neonatal lethality in argininosuccinate synthetase-deficient mice. Kok CY, Cunningham SC, Carpenter KH, Dane AP, Siew SM, Logan GJ, Kuchel PW, Alexander IE. Mol Ther. 2013 Oct;21(10):1823-31. doi: 10.1038/mt.2013.139. Epub 2013 Jul 2. PMID: 23817206.
  3. Gene therapy researchers' assessments of risks and perceptions of risk acceptability in clinical trials. Deakin CT, Alexander IE, Hooker CA, Kerridge IH. Mol Ther. 2013 Apr;21(4):806-15. doi: 10.1038/mt.2012.230. Epub 2013 Jan 22. PMID: 23337985.
  4. Gene therapy for tolerance: high-level expression of donor major histocompatibility complex in the liver overcomes naive and memory alloresponses to skin grafts. Cunningham EC, Tay SS, Wang C, Rtshiladze M, Wang ZZ, McGuffog C, Cubitt J, McCaughan GW, Alexander IE, Bertolino P, Sharland AF, Bowen DG, Bishop GA. Transplantation. 2013 Jan 15;95(1):70-7. doi: 10.1097/TP.0b013e318278d39a. PMID: 23263501.
  5. Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders. Alexander IE, Kok C, Dane AP, Cunningham SC. J Inherit Metab Dis 2012 Jul;35(4):641-5. Epub 2012 Mar 9. PMID 22403018.
  6. Mitochondrial Respiratory Chain Hepatopathies: Role of Liver Transplantation. A Case Series of Five Patients. De Greef E, Christodoulou J, Alexander IE, Shun A, O’Loughlin EV, Thorburn DR, Jermyn V, Stormon MO. J. Inher. Meta. Dis. Reports 2012 4:5-11. PMID 23430890.
  7. A murine model of neurofibromatosis type 1 pseudarthrosis featuring proliferative fibrous tissue and osteoclast-like cells. El-Hoss J. Sullivan K, Cheng T, Yu NYC, Bobyn JD, Peacock L, Mikulec K, Baldock P, Alexander IE, Schindeler A, Little DG. J. Bone Miner. Res. 2012 27(1):68-78, Epub 28th Sept 2011. PMID 21956219.
  8. Gene therapy: progress in childhood disease. Ginn SL, Alexander IE. J Paediatr Child Health, 2012 Jun;48(6):466-71. Epub 2011 Oct 21. PMID 22017270.
  9. Adeno-associated virus vectors: immunobiology and potential use for immune modulation. Logan GJ, Alexander IE. Curr Gene Ther. 2012 Aug 1;12(4):333-43. PMID 22856608.
  10. Comparison of gene transfer to the murine liver following intra-peritoneal and intra-portal delivery of hepatotropic AAV pseudo-serotypes. Dane AP, Wowro SJ, Cunningham SC, Alexander IE. Gene Therapy 2013 Apr;20(4):460-4. Epub 16th August 2012. PMID 22895507.
  11. Human induced pluripotent stem cells derived under feeder-free conditions display unique cell cycle and DNA replication gene profiles. Chung HC, Lin RC, Logan GJ, Alexander IE, Sachdev PS, Sidhu KS. Stem Cells Dev. 2012 Jan 20;21(2):206-16. Epub 2011 Jun 1. PMID 21506733.
  12. Study of LPIN1, LPIN2 and LPIN3 in rhabydomyolysis and exercise-induced myalgia. Michot C., Hubert L., Romero N., Gouda A., Mathew S., Kirk E., Viollet L., Rahman S., Bekri S., Peters H., McGill J., Glamuzina E., Hagen M., Alexander I., Kirmse B., Laforet P., Benlian P., Munnich A., JeanPierre M., Elpeleg O., Ophry P., Delahodde A, de Keyzer Y. and de Lonlay P. Journal of Inherited Metabolic Disease 2012 35(6):1119-1128, Epub 6th April 2012. PMID 22481384.
  13. Gene Therapy for tolerance: High-level expression of donor MHC in the liver overcomes naïve and memory alloresponses to skin grafts. Cunningham E.C., Tay S.S., Wang C., Rtshiladze M., Wang Z.Z., McGuffog C., Cubitt J., McCaughan G.W., Alexander I.E., Bowen D., Sharland A.F., Bertolino P.D. and Bishop G.A. Transplantation 2012 95(1):70-77, Epub December 2012. PMID 23263501.


Key Publications

  1. Limiting {gamma}c expression differentially affects signaling via the interleukin (IL)-7 and IL-15 receptors. Smyth CM, Ginn SL, Deakin CT, Logan GJ, Alexander IE. Blood 2007, 110: 91-8. PMID: 17363735.
  2. Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia. Ginn SL, Curtin JA, Kramer B, Smyth C, Wong M, Kakakios A, McCowage G, Watson D, Alexander SI, Latham M, Cunningham S, Zheng M, Hobson L, Rowe PB, Fischer A, Cavazzana-Calvo M, Hacein-Bey-Abina S and Alexander IE. Med. J. Aust. 2005, 182: 458-463. PMID: 15865589.
  3. Fibroblasts can be genetically modified to produce excitable cells capable of electrical coupling. Kizana E. Ginn SL, Allen DG, Ross DL and Alexander IE. Circulation 2005, 111: 394-398. PMID: 15687125.
  4. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 [gc deficiency]. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A and Cavazzana-Calvo M. Science 2003, 302: 415-419. PMID: 14564000.
  5. Kojima Y ., Kaufman-Francis K., Studdert J.B., Steiner K.A., Power M.D., Loebel D.A.F., Jones V., Hor A., de Alencastro G., Logan G., Teber E.T., Tam O.H., Stutz M.D., Alexander I.E., Pickett H.A. and Tam P.P.L. (2014) The transcriptional and functional properties of mouse epiblast stem cells resemble the anterior primitive streak. Cell Stem Cells 13:1-14, epub 17th October, 2013. IF 22.151
  6. Lisowski L., Dane A.P., Chu. K., Zhang Y., Cunningham S.C., Wilson E.M., Nygaard S., Grompe, M., Alexander I.E. and Kay M.A. (2014) Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature 506(7488):382-386, epub 25th December, 2013. IF 42.351

Review Articles

  1. Gene therapy clinical trials worldwide to 2012 - an update. Ginn SL, Alexander IE, Edelstein ML, Abedi MR, Wixon J. J Gene Med. 2013 Feb;15(2):65-77. doi: 10.1002/jgm.2698. Review. PMID: 23355455.
  2. The ethics of gene therapy: balancing the risks. Deakin CT, Alexander IE, Kerridge I. Curr. Opin. Mol. Ther. 2010 12: 578-85. PMID: 20886390.
  3. Accepting risk in clinical research: is the gene therapy field becoming too risk-averse? Deakin CT, Alexander IE, Kerridge I. Mol. Ther. 2009, 17: 1842-8. PMID: 19773741.
  4. Gene therapy in transplantation. Laurence JM, Allen RD, McCaughan GW, Logan GJ, Alexander IE, Bishop GA, Sharland AF. Transplant Rev (Orlando)2009, 23: 159-70. PMID: 19428235.
  5. Potential of AAV vectors in the treatment of metabolic disease. Alexander IE, Cunningham SC, Logan GJ, Christodoulou J. Gene Ther. 2008, 15: 831-9. PMID: 18401432.