Biallelic genome editing of human iPS cells for functional studies and disease modeling
Human pluripotent stem cells are an ideal model system to study basic cellular and developmental processes in a normal diploid cell. The advent of site-specific nucleases and improved conditions for human iPSC culture now permits efficient engineering of human stem cells. CRISPR-Cas9 technology, in particular, provides a facile tool for the generation of biallelic mutations, thus empowering functional studies of human genes in a model human cell. In my talk, I will present strategy that provides an efficient, rapid, and scalable method for conditional and reversible gene knockouts in diploid or aneuploid cells such as pluripotent stem cells, 3D organoids and cell lines (Nature Methods, 2017). Other new development of genome-editing technologies in my lab will also be disucssed.