10:00-17:40
March 21, 2024
| GCT-Virtual March 21, 2024 Program |
|
| 10:00:10:20 | Artificial RNA Editing toward Treatment for Diseases Caused by Point Mutation |
| Toshifumi Tsukahara, Japan Advanced Institute of Science and Technology, Japan | |
| 10:20:10:40 | Study on the mechanism of selenoprotein O targeting JAK1 to induce the release of macrophage external net to regulate the occurrence of ulcerative colitis in mice |
| Xu Shi, Northeast Agricultural University, China | |
| 10:40:11:00 | Generation of a bank of clinical-grade, HLA homozygous iPSC lines with high coverage of the Spanish population |
| Bernd Kuebler, Institut d´Investigació Biomédica de Bellvitge (IDIBELL), L’Hospitalet de Llobregat , Spain | |
| 11:00:11:20 | A cross-correction mechanism enhances gene therapy efficacy in a mouse model of CDKL5 deficiency disorder and patient-derived cortical organoids |
| Giorgio Medici, University of Bologna, Italy | |
| 11:20:11:40 | Increased telomerase improves motor function and alpha-synuclein pathology in a transgenic mouse model of Parkinson’s disease associated with enhanced autophagy |
| Gabriel Saretzki, Biosciences Institute, Newcastle University, UK | |
| 11:40:12:00 | |
| 12:00:12:10 Networking Break |
|
| 12:10:12:30 | Perspective of DMPK on rAAV-Based Gene Therapy |
| Nancy Chen, Takeda Inc., USA | |
| 12:30:12:50 | CRISPR/Cas9-based disease modelling and functional correction of Interleukin 7 Receptor alpha Severe Combined Immunodeficiency in T-lymphocytes and hematopoietic stem cells |
| Rajeev Rai, Great Ormond Street Institute of Child Health, University College London, UK | |
| 12:50:13:10 | DNMT1-Specific Covalent Tracking Of DNA Methylation In Mouse Embryonic Cells |
| Vaidotas Stankevicius, Institute of Biotechnology, Life Sciences Center, Vilnius University, Lithuania | |
| 13:10:13:30 | A method for multiplexed full-length single-molecule sequencing of the human mitochondrial genome |
| Ivo Gut, Centro Nacional de Análisis Genómico (CNAG), Barcelona, Spain | |
| 13:30:13:50 | Evaluation of human adipose-derived mesenchymal stromal cell Toll-like receptor priming and effects on interaction with prostate cancer cells |
| Marxa L. Figueiredo, Purdue University, USA | |
| 13:50:14:10 | Assessing the Presence and Severity of GT-Induced Neuropathies in Juvenile Rodents and Primates based on Preclinical Nerve Conduction (NC) |
| Monica R. Metea, Preclinical Electrophysiology Consulting, MA, USA | |
| 14:10:14:20 Networking Break |
|
| 14:20:14:40 | Successfully Navigating the FDA Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The NIH Platform Vector Gene Therapy Experience |
| Richa Madan Lomash, Therapeutic Development Branch, NCATS, NIH, USA | |
| 14:40:15:00 | Genome editing without nucleases confers proliferative advantage to edited hepatocytes and corrects Wilson disease |
| Jay (Chih-Chieh) Wang, Alexion Genomic Medicine, Astra Zeneca Rare Disease, MA,USA | |
| 15:00:15:20 | Encapsulate the components of CRISPR/Cas9 into extracellular vesicles by protein palmitoylation |
| Yaoyao Lu, Centre de Recherche du CHU de Québec -Université Laval, Canada | |
| 15:20:15:40 | Co-transplantation of mesenchymal stem cells and endothelial cells with islet grafts: A strategy to improve post-tx engraftment of pancreatic islets |
| Raza Ali Naqvi, Department of Periodontics, University of Illinois, USA | |
| 15:40:16:00 | Cell-based gene therapy for neuroblastoma |
| Natarajan Aravindan, Oklahoma State University, USA | |
| 16:00:16:20 | Cell specific tumor targeted peptide therapeutics for neuroblastoma |
| Dinesh Babu Somasundaram, Oklahoma State University, USA | |
| Closing Remarks | |