Thursday, 14th November ASSCR, AGCTS, ISCT ANZ and Friends Joint Scientific Conference 2019

Stem cells and genome engineering: the perfect match to support the current surge in biomedical sciences

Presented by: Dr Dmitry A. Ovchinnikov

• Genome Editing for Duchenne Muscular Dystrophy - Charles A Gersbach
• High-throughput selection of novel AAV capsids in a murine xenograft liver model: towards next generation of human liver-tropic AAV variants - Renina G.O. Navarro
• Generation of novel cardiac specific AAV vectors by directed evolution in human iPSC derived cardiomyocytes - Cindy Kok
• Nature trumps nurture: Use of naturally liver-tropic adeno-associated viruses as vector platforms for liver-directed human gene therapy - Claus V Hallwirth
• Next generation of gene therapy vectors for clinical applications: unlocking the hepatotropic potential of prototypical AAV2 - Marti Cabanes-Creus

• Developing safe and allo-tolerated pluripotent stem cells - Natalie L Payne
• The immunosuppressive capacity of mesenchymal stromal cells is conferred by their apoptosis in vivo - Tracy Heng
• Antisense oligonucleotide mediated knockdown of RNA-binding proteins SAM68 and hnRNP-A1 to modulate expression of the survival motor neuron 2 gene. - Ianthe L Pitout
• Liver-specific Delivery of GalNac-siRNA Targeting TMPRSS6 Ameliorates Ineffective Erythropoiesis and Anaemia in β-Thalassaemia mice - Jim Vadolas
• New phosphoinositide-3 kinase-based gene therapies for the failing heart - Julie McMullen

• Rapid administration of third-party cytomegalovirus (CMV), Epstein-Barr virus (EBV) or adenovirus (Adv) specific T-cells (VSTs) post-haemopoietic stem cell transplant (HSCT) - Wei Jiang
• Neuregulin 1 promotes intestinal stem cell proliferation and improves tissue regeneration following damage - Thierry Jarde
• DISTINCT EARLY EMBRYONIC PREIMPLANTATION DEVELOPMENTAL PROGRAMS DRIVE REPROGRAMMING INTO PRIMED AND NAIVE INDUCED PLURIPOTENCY - Xiaodong Liu
• Towards retinal regeneration: Using CRISPR activation to promote direct reprogramming of retinal glial cells to photoreceptors - Raymond Wong
• Adoptive Cellular Immunotherapy for Post-transplant Viral Complications - Rajiv Khanna

• The use of endogenously expressed microRNA for targeted gene delivery to cancer. - Jason C Steel
• Development of a splice-switching molecular therapy for SOD1 motor neurone disease - Loren L Flynn
• Comparative analysis of the heterogeneity of iPSC-derived and tissue-derived MSCs - Margeaux Hodgson-Garms
• Antisense Oligonucleotide-Mediated Exon Skipping to Treat Spinocerebellar Ataxia Type 3   - Craig S McIntosh
• RNA therapeutics for cancer: are we there yet? - Peter Leedman

• Modulation of CNOT3 expression to treat retinitis pigmentosa 11 using antisense oligomers - Sue Fletcher
• Generating sperm from XX individuals - Takayuki Hirota
• Gene-editing in human pluripotent stem cells for disease modelling and testing therapies in the retinal dystrophies - Robyn V Jamieson
• The Vector and Genome Engineering Facility (VGEF): Customized service for your research needs - Predrag Kalajdzic
• Meise System Autologous Serum Eye Drops Validation - Michelle W Keir
• Differentiation and proliferation characteristics of tonsil-derived mesenchymal stem cells according to age, gender and tissue source - Han Su Kim
• EVALUATING THE EFFECT OF MONOMERIC AMYLOID β ON HEALTH OF FOREBRAIN NEURONS DERIVED FROM HUMAN EMBRYONIC STEM CELLS - Eunbi Kwak
• AAV vector gene delivery of DNase I in lupus-prone mice doesn't improve renal function but does decrease C3/IgG renal deposition - Grant J Logan
• In vitro modelling of SCN2A genetic epilepsy using human pluripotent stem cells - Cristiana Mattei
• Utilising patient-specific retinal organoids to investigate the role of SNRNP200 variants of unknown significance in severe early onset retinitis pigmentosa - Carla Mellough
• Proline induces neural differentiation and promotes embryo development in a growth factor-like manner - Michael B. Morris
• Transcriptomic data of human mesenchymal stem cells secretome and targets for wound healing - Huan Ting Ong
• Profound impact of APOE4 in human iPSC-derived microglia-like cells compared to familial Alzheimer’s disease mutations - Lezanne Ooi
• Wound healing promoting activity of tonsil-derived stem cells on 5-fluorouracil-induced oral mucositis model - Hae Sang Park
• Identification and Expression of Islet-enriched microRNAs - Vijit Saini
• Identifying AAV integration sites in next-generation sequencing data - Suzanne Scott
• Combinations of Promoters and Virus Capsids May Affect Gene Transduction Efficiency in Adeno-associated Virus - Yoshihide YS Sehara
• L-proline acts like a growth factor to drive the differentiation of mESCs to neurectoderm via embryologically relevant cell populations   - Rachel A Shparberg
• Understanding the role of WNT signalling in generating midbrain dopaminergic neurons of the substantia nigra pars compacta /A9 subtype neurons - Shanti Sibuea
• Antigen-encoding bone marrow transfer deletes mature antigen-specific B cells in recipients and inhibits antigen-specific antibody production - Raymond J Steptoe
• Defining altered Cl^- conductance and phagocytosis in retinal pigment epithelium containing an F305S BEST1 mutation associated with Best disease - Szeifoul Szeifoul
• The Impact of Science Communication on Public Attitude towards Human Genome Editing. - Tamara Treleaven
• Defining protective responses of human micro-lenses to cataract initiating factors - Chitra Umala Dewi
• Advancing the therapeutic potential of lentiviral β-globin gene therapy with selective decrease of α2-globin mRNA expression - Jim Vadolas
• A novel pixel-clique statistical model for probing subcellular FRET efficiency of Rho-GTPases using live cell imaging - Liyuan Wang
• SCF Promotes Proliferation during Definitive Endoderm Differentiation of Human Pluripotent Stem Cells - Tianyu WANG
• Developing a methodological infrastructure for the qualitative and quantitative evaluation of unstable genomic elements in rAAV vectors - Patrick Wilmott
• Comparison of Aerobic and Anaerobic bacteria, yeast and mould detection using the BacT/Alert® 3D™and Virtuo™ systems for Haematopoietic Progenitor Cells - Karieshma Kabani
• Mutation characterisation in patients with Usher syndrome type 2A and application of therapeutic alternative splicing - Khine Zaw
• Making the inactive active through changes in antisense oligomer chemistries - Khine Zaw
• Atherogenic ox-LDL results in human endothelial progenitor depletion and dysfunction through pathological endothelial-to-mesenchymal transition - Jilai Zhao
• Targeting adeno-associated viral AAV vectors for systemic delivery to the skeleton - Lucinda R Lee