A human ESC line for efficient CRISPR editing of pluripotent stem cells

Elad Sintov; Dario Gerace; Douglas A Melton

doi:10.1016/j.scr.2021.102591

A human ESC line for efficient CRISPR editing of pluripotent stem cells

Stem Cell Res. 2021 Dec:57:102591. doi: 10.1016/j.scr.2021.102591. Epub 2021 Nov 2.

Authors

Elad Sintov¹, Dario Gerace², Douglas A Melton³

Affiliations

¹ Department of Stem Cell and Regenerative Biology, Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA. Electronic address: sintov@fas.harvard.edu.
² Department of Stem Cell and Regenerative Biology, Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA.
³ Department of Stem Cell and Regenerative Biology, Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA; Howard Hughes Medical Institute, Chevy Chase, MD 20815, USA. Electronic address: dmelton@harvard.edu.

PMID: 34741877
DOI: 10.1016/j.scr.2021.102591

Abstract

Human pluripotent stem cells (hPSC) can be directed to differentiate in vitro into insulin-prorducing beta cells (SC-β). Although these cells accurately respond to glucose and can reverse diabetes in preclinical models improvments in the final cell products are desirable. For example, safety, controlling the cellular compositions and protection against immune rejection may be addressed by genetic modifications of SC-β pre-transplantation. To screen for gene targets, we have generated a human embryonic stem cell line (hESC) that constitutively express the enhanced specificity Streptococcus pyogenes Cas9 (eSpCas9) gene, knocked-in into the GAPDH locus.