Moving Us Forward

This session will focus on the critical role of optical genome mapping (OGM) in assessing the genome integrity and off-target events in cells engineered for therapy development, specifically within the context of stem cell research. The presentation will begin with an introduction by Alicia Bertolotti from Bionano, who will highlight how OGM provides a high resolution, genome-wide, sensitive, and reproducible workflow that can be applied in cell and gene therapy applications. Following the introduction, Professor Emeritus Dr. Jeanne Loring from the Scripps Research Institute will then share her expertise on iPSC culture and genome engineering. Her talk will focus on the use of OGM to assess the genomic stability and safety of iPSCs—crucial for their use in disease modeling, drug testing, and potentially therapeutic applications. Finally, Dr. Lucia Gallego from the Department of Molecular Biochemistry at Ruhr-University of Bochum will present findings from their recent publication. Their study leverages the CRISPR/Cas9 gene-editing technology in human induced pluripotent stem cells (hiPSCs) to create cerebral organoids. Overall, this session highlights the necessity of using high-resolution, unbiased genome-wide analysis workflow of OGM to assess the safety and efficacy of gene-edited models, propelling forward the field of cell and gene therapy research.