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!


Dr Samantha Ginn: Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes. (2020). Journal of Hepatology Reports


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


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


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


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


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


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


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


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


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


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


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

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