Gene Therapy Unit Publications

Key Publications

1. Smyth CM, Ginn SL, Deakin CT, Logan GJ, Alexander IE. (2007) Limiting {gamma}c expression differentially affects signaling via the interleukin (IL)-7 and IL-15 receptors. Blood, 110: 91-8.
   PMID: 17363735
2. 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. (2005) Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia. Med. J. Aust., 182: 458-463.
   PMID: 15865589
3. Kizana E. Ginn SL, Allen DG, Ross DL and Alexander IE. (2005) Fibroblasts can be genetically modified to produce excitable cells capable of electrical coupling. Circulation, 111: 394-398.
   PMID: 15687125
4. 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. (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 [gc deficiency]. Science, 302: 415-419.
   PMID: 14564000
5. Fleming J, Ginn SL, Weinberger RP, Trahair TN, Smythe JA and Alexander IE. (2001) Adeno-associated virus and lentivirus vectors mediate efficient and sustained transduction of cultured mouse and human dorsal root ganglia sensory neurons. Hum. Gene Ther., 12: 77-86.
   PMID: 11177545

Review Articles

1. Deakin CT, Alexander IE, Kerridge I. (2010) The ethics of gene therapy: balancing the risks. Curr. Opin. Mol. Ther., 12: 578-85.
   PMID: 20886390
2. Deakin CT, Alexander IE, Kerridge I.  (2009) Accepting risk in clinical research: is the gene therapy field becoming too risk-averse?  Mol. Ther., 17: 1842-8.
   PMID: 19773741
3. Laurence JM, Allen RD, McCaughan GW, Logan GJ, Alexander IE, Bishop GA, Sharland AF.  (2009) Gene therapy in transplantation.  Transplant Rev (Orlando), 23: 159-70.
   PMID: 19428235
4. Alexander IE, Cunningham SC, Logan GJ, Christodoulou J.  (2008) Potential of AAV vectors in the treatment of metabolic disease.  Gene Ther., 15: 831-9.
   PMID: 18401432
5. Lee VW, Wang YM, Wang YP, Zheng D, Polhill T, Cao Q, Wu H, Alexander IE, Alexander SI, Harris DC.  (2008) Regulatory immune cells in kidney disease.  Am. J Physiol. Renal Physiol, 295: F335-42.
   PMID: 18417542

Recent Publications

1. Chung HC, Lin RC, Logan GJ, Alexander IE, Sachdev P and Sidhu KS (2011). Human induced pluripotent stem cells derived under feeder-free conditions display unique cell cycle and DNA replication gene profiles. Stem Cells Dev., Epub Apr 20.
   PMID: 21506733
2. Cunningham SC, Kok CY, Dane AP, Carpenter K, Kizana E, Kuchel PW and Alexander IE (2011). Induction and Prevention of Severe Hyperammonemia in the spf(ash) Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery.  Mol. Ther., Epub Mar 8.
   PMID: 21386824
3. Sharland A, Logan GJ, Bishop A, Alexander IE.  (2010) Liver-directed gene expression using recombinant AAV 2/8 vectors -- a tolerogenic strategy for gene delivery?  Discov. Med., 9: 519-27.
   PMID: 20587341
4. Ginn SL, Liao SH, Dane AP, Hu M, Hyman J, Finnie JW, Zheng M, Cavazzana-Calvo M, Alexander SI, Thrasher AJ, Alexander IE.  (2010 ) Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and gammac Overexpression.  Mol. Ther., 18: 868-70.
   PMID: 20354504
5. Logan GJ, Wang L, Zheng M, Coppel RL, Alexander IE.  (2009) Antigen fusion with C3d(3) augments or inhibits humoral immunity to AAV genetic vaccines in a transgene-dependent manner.  Immunol. Cell Biol., 88: 228-32.
   PMID: 19935770
6. Dane AP, Cunningham SC, Graf NS, Alexander IE.  (2009) Sexually Dimorphic Patterns of Episomal rAAV Genome Persistence in the Adult Mouse Liver and Correlation With Hepatocellular Proliferation.  Mol. Ther., 17:1548-54.
   PMID: 19568224
7. Cunningham SC, Spinoulas A, Carpenter KH, Wilcken B, Kuchel PW, Alexander IE.  (2009) AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spf(ash) Mice.  Mol. Ther., 17:1340-6.
   PMID: 19384294
8. Ginn SL, Cunningham SC, Zheng M, Spinoulas A, Carpenter KH, Alexander IE.  (2009) In vivo assessment of mutations in OTC for dominant-negative effects following rAAV2/8-mediated gene delivery to the mouse liver.  Gene Ther., 16: 820-3.
   PMID: 19357713
9. Liu R, Ginn SL, Lek M, North KN, Alexander IE, Little DG, Schindeler A.  (2009) Myoblast sensitivity and fibroblast insensitivity to osteogenic conversion by BMP-2 correlates with the expression of Bmpr-1a.  BMC Musculoskelet. Disord., 10: 51.
   PMID: 19442313
10. Lee AS, Kahatapitiya P, Kramer B, Joya JE, Hook J, Liu R, Schevzov G, Alexander IE, McCowage G, Montarras D, Gunning PW, Hardeman EC.  (2009) Methylguanine DNA methyltransferase-mediated drug resistance-based selective enrichment and engraftment of transplanted stem cells in skeletal muscle.  Stem Cells, 27: 1098-108.
    PMID: 19415780  

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