CRISPR/Cas9 genome editing in human pluripotent stem cells: Harnessing human genetics in a dish

摘要: Because of their extraordinary differentiation potential, human pluripotent stem cells (hPSCs) can differentiate into virtually any cell type the body, providing a powerful platform not only for generating relevant types useful replacement therapies, but also modeling development and disease. Expanding this structures resembling organs, termed organoids, have been recently obtained from hPSCs through tissue engineering. Organoids exhibit multiple self-organizing recapitulating in part physiology cellular interactions observed organ vivo, offering unprecedented opportunities disease modeling. To fulfill promise, engineering needs to be supported by robust scalable genome editing technologies. With advent CRISPR/Cas9 technology, manipulating has now become an easy task, allowing modifying with superior precision, speed, throughput. Here we review current potential applications technology how they contribute establish as model choice studying genetics. Developmental Dynamics 245:788-806, 2016. © 2016 Wiley Periodicals, Inc.