Awards
Best Paper Prize
Best paper prizes are awarded twice each year to first or corresponding member authors (original research papers only). Watch your email for the next round and submit your latest exciting publication! The winning publications are listed below – congrats to all!
2020
Dr Samantha Ginn: Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes. (2020). Journal of Hepatology Reports https://doi.org/10.1016/j.jhepr.2019.100065
2019
Professor Ann Simpson: Ex Vivo Expansion of Murine MSC Impairs Transcription Factor-Induced Differentiation into Pancreatic b-Cells. (2019). Stem Cells International Article ID 1395301
Dr Chandana Herath: Liver-Targeted Angiotensin Converting Enzyme 2 Therapy Inhibits Chronic Biliary Fibrosis in Multiple Drug-Resistant Gene 2-Knockout Mice. (2019). Hepatology Communications DOI 10.1002/hep4.1434
Dr May Aung-Htut: Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides. (2019). Scientific Reports https://doi.org/10.1038/s41598-019-49385-6
2018
Dr Livia Carvalho: Synthetic adeno-associated viral vector efficiently targets mouse and nonhuman primate retina in vivo. (2018). Human Gene Therapy 29(7): 771-784
Dr George von Jonquieres: Uncoupling N-acetylaspartate from brain pathology: implications for Caravan disease gene therapy. (2018). Acta Neuropathology 135(1): 95-113
2017
Dr Samantha Ginn: Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency. (2017). Molecular Therapy – Nucleic Acids 6: 1-14
Dr Grant Logan: Identification of liver-specific enhancer-promoter activity in the 3’ untranslated regions of the wild-type AAV2 genome. (2017). Nature Genetics 49(8): 1267-1273
2016
Astrid Glaser: GFP to BFP Conversion: A Versatile Assay for the Quantification of CRISPR/Cas9-mediated Genome Editing. (2016). Molecular Therapy – Nucleic Acids 5(7): e334
Professor Elizabeth Rakoczy: Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial. (2015). Lancet 386(10011): 2395-2403
Dr Binhai Ren: Pancreatic Transdifferentiation and Glucose-Regulated Production of Human Insulin in the H4IIE Rat Liver Cell Line. (2016). International Journal of Molecular Sciences 17(4): 534
2015
Dr Paul Gregorevic: Development of Novel Activin-Targeted Therapeutics. (2015). Molecular Therapy 23(3): 434–444
Professor Ann Simpson: Reversal of diabetes following transplantation of an insulin-secreting human liver cell line: Melligen cells. (2015). Molecular Therapy — Methods & Clinical Development 2: 15011
2014
A/Prof Frank Alderuccio: Gene therapy delivery of myelin oligodendrocyte glycoprotein (MOG) via hematopoietic stem cell transfer induces MOG-specific B cell deletion. (2014). Journal of Immunology 192(6): 2593-2601
Dr Claire Deakin: Impact of next-generation sequencing error on analysis of barcoded plasmid libraries of known complexity and sequence. (2014). Nucleic Acids Research 42(16): e129
Kane Greer: Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene. (2014). Molecular Therapy – Nucleic Acids 3: e155
2013
A/Prof Glen Reid: Restoring expression of miR-16: a novel approach to therapy for malignant pleural mesothelioma. (2013). Annals of Oncology 24(12): 3128-3135
Dr Binhai Ren: Long-term reversal of diabetes in non-obese diabetic mice by liver-directed gene therapy. (2013). Journal of Gene Medicine 15(1): 28-41
Professor Geoff Symonds: Pre-clinical Safety and Efficacy of an Anti-HIV-1 Lentiviral Vector Containing a Short Hairpin RNA to CCR5 and the C46 Fusion Inhibitor. (2014). Molecular Therapy — Methods & Clinical Development 1: 11
Dr Georg von Jonquieres: Glial Promoter Selectivity following AAV-Delivery to the Immature Brain. (2013). PLoS One8(6): e65646
2012
Professor Sue Fletcher: Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene. (2012). Molecular Therapy – Nucleic Acids 1: e48
Dr Prue Plummer: MicroRNAs regulate tumor angiogenesis modulated by endothelial progenitor cells. (2013). Cancer Research 73(1): 341-352
A/Professor Frank Alderuccio: Nonmyeloablative Conditioning Generates Autoantigen-Encoding Bone Marrow That Prevents and Cures an Experimental Autoimmune Disease. (2012). American Journal of Transplantation 12(8): 2062-2071
Dr Christopher Siatskas: Thymic Gene Transfer of Myelin Oligodendrocyte Glycoprotein Ameliorates the Onset but Not the Progression of Autoimmune Demyelination. (2012). Molecular Therapy 20(7): 1349-1359
2011
Dr Sharon Cunningham: Induction and Prevention of Severe Hyperammonemia in the spf ash Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery. (2011). Molecular Therapy (19)5: 854–859
Dr Michael Gantier: Analysis of microRNA turnover in mammalian cells following Dicer1 ablation (2011). Nucleic Acids Research 39(13): 5692-703
Dr Shannen Lau: Enhanced Extravasation, Stability and in Vivo Cardiac Gene Silencing via in Situ siRNA−Albumin Conjugation. (2012). Molecular Pharmaceutics 9(1): 71-80
2010
Dr Samantha Ginn: Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and gc Overexpression. (2010). Molecular Therapy 18(5): 965-976
Dr Albert Mellick: Using the Transcription Factor Inhibitor of DNA Binding 1 to Selectively Target Endothelial Progenitor Cells Offers Novel Strategies to Inhibit Tumor Angiogenesis and Growth. (2010). Cancer Research 70(18): 7273-7282
Professor Elizabeth Rakoczy: rAAV.sFlt-1 Gene Therapy Achieves Lasting Reversal of Retinal Neovascularization in the Absence of a Strong Immune Response to the Viral Vector. (2010). Investigative Ophthalmology & Visual Science 50(9):4279-4287
Professor Steve Wilton: Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations. (2010). Neuromuscular Disorders 20(12): 810-816
2009
Dr Sharon Cunningham: AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spf ashMice. (2009). Molecular Therapy 17(8): 1340-1346
Dr Allison Dane: Sexually Dimorphic Patterns of Episomal rAAV Genome Persistence in the Adult Mouse Liver and Correlation with Hepatocellular Proliferation. (2009). Molecular Therapy 17(9): 1548-1554
Dr Aparajita Khatri: Cytosine Deaminase-Uracil Phosphoribosyltransferase and Interleukin (IL)-12 and IL-18: A Multimodal Anticancer Interface Marked by Specific Modulation in Serum Cytokines. (2009). Clinical Cancer Research15(7): 2323-2334
Dr Grant Logan: Antigen-specific humoral tolerance or immune augmentation induced by intramuscular delivery of adeno-associated viruses encoding CTLA4-Ig-antigen fusion molecules. (2009). Gene Therapy 16(2): 200-210