Innovation Showcase
Human induced pluripotent stem cells (hiPSCs) and genome engineering technologies have become powerful in vitro tools to model and study various genetic conditions. In this session, Prof. Bill Skarnes, a recognized leader in stem cell genome engineering and Director of Cellular Engineering at the Jackson Laboratory for Genomic Medicine, will present on 1) the efforts undertaken that led to the identification of KOLF2.1J as a suitable reference hiPSC line, 2) provide updates on the generation of hundreds of variants in KOLF2.1J using high-throughput precision editing as part of the NIH-funded iPSC Neurodegenerative Disease Initiative (iNDI), and 3) introduce the MorPhiC project that aims to develop a catalog of molecular and cellular phenotypes for null alleles for every human gene. In the second half of this session, Dr. Camille Januel, from the Chan Zuckerberg Biohub San Francisco, will discuss her efforts using CRISPR technology to tag organelles with the aim of studying organelle dynamics in various hiPSC-derived cell types. This platform will enable the detailed examination of organelles via live cell imaging and proteomics to better understand cellular impacts of disease-associated mutations.