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Updated: Oct 14, 2025
Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
Published on: February 2, 2016
A human ESC line for efficient CRISPR editing of pluripotent stem cells
Elad Sintov1, Dario Gerace1, Douglas A Melton2
1Department of Stem Cell and Regenerative Biology, Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA.
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.
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