March 21, 2024

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
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
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