Dev Dyn. 2016 Jul;245(7):788-806. doi: 10.1002/dvdy.24414. Epub 2016 Jun 09.
CRISPR/Cas9 genome editing in human pluripotent stem cells: Harnessing human genetics in a dish.
Developmental dynamics : an official publication of the American Association of Anatomists
Federico González
Affiliations
Affiliations
- Institute for Bioengineering of Catalonia (IBEC), Calle Baldiri Reixac 15-21, 08028, Barcelona, Spain.
PMID: 27145095
DOI: 10.1002/dvdy.24414
Abstract
Because of their extraordinary differentiation potential, human pluripotent stem cells (hPSCs) can differentiate into virtually any cell type of the human body, providing a powerful platform not only for generating relevant cell types useful for cell replacement therapies, but also for modeling human development and disease. Expanding this potential, structures resembling human organs, termed organoids, have been recently obtained from hPSCs through tissue engineering. Organoids exhibit multiple cell types self-organizing into structures recapitulating in part the physiology and the cellular interactions observed in the organ in vivo, offering unprecedented opportunities for human disease modeling. To fulfill this promise, tissue engineering in hPSCs needs to be supported by robust and scalable genome editing technologies. With the advent of the CRISPR/Cas9 technology, manipulating the genome of hPSCs has now become an easy task, allowing modifying their genome with superior precision, speed, and throughput. Here we review current and potential applications of the CRISPR/Cas9 technology in hPSCs and how they contribute to establish hPSCs as a model of choice for studying human genetics. Developmental Dynamics 245:788-806, 2016. © 2016 Wiley Periodicals, Inc.
© 2016 Wiley Periodicals, Inc.
Keywords: CRISPR/Cas9; disease modeling; human genetics; human pluripotent stem cells; tissue and genome engineering
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