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

被引：2
|
作者：
Sintov, Elad [1 ]
Gerace, Dario [1 ]
Melton, Douglas A. [1 ,2 ]
机构：
[1] Harvard Univ, Dept Stem Cell & Regenerat Biol, Harvard Stem Cell Inst, Cambridge, MA 02138 USA
[2] Howard Hughes Med Inst, Chevy Chase, MD 20815 USA
来源：
STEM CELL RESEARCH | 2021年 / 57卷
关键词：
D O I：
10.1016/j.scr.2021.102591
中图分类号：
Q813 [细胞工程];
学科分类号：
摘要：
Human pluripotent stem cells (hPSC) can be directed to differentiate in vitro into insulin-prorducing beta cells (SC-beta). 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-beta 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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页数：4
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