Table 1.

Summary of CRISPR/Cas9 Utilized in Human Stem Cell Research

	Cell Type	Function	Year	References
Basic Biological Studies
Gene knockout	ESCs	Mettl3erase m6A modification leading to cell differentiation	2014	13
	ESCs	p53/63/73 knockout causes mesendodermal differentiation	2017	14
Control expression	ESCs	downregulation of Oct4 leads to lineage differentiation	2014	18
	iPSCs	Dox-inducible CRISPRi represses gene expression	2016	19
	PSCs	Dox-inducible CRISPRa activates gene expression	2017	20
	NSCs	inhibition of miR-199a/214 increases tumor tropism	2016	23
Genome scale screening	ESCs	identification of essential genes for cell survival by knockout of 18,080 genes simultaneously	2014	28
	iPSCs	identification of 326 functional loci of lncRNAs	2017	29
Gene knockin	iPSCs	generation of collagen-GFP reporter for cell sorting	2016	35
	ESCs	monitor pharmacological profiles of striatal cultures using DARPP-32-GFP reporter cell	2017	36
Clinical Translational Studies
Disease model and drug screening	ESCs	RB1-null cells showed aberrant mitochondria and were sensitive to carboplatin (retinoblastoma)	2017	40
	iPSCs	isogenic iPSCs model for pathomechanism and drug screening (myelodysplastic syndrome)	2016	42
	iPSCs	point mutation iPSCs model for studying individual difference in hypoglycemia	2017	43
	ESCs	WRN-null hESCs model Werner syndrome (WS-iPSCs showed abnormal karyotypes seriously)	2015	44
	organoids	gene knockout kidney organoids showed cyst of tubules	2015	45
	organoids	modeling dyskeratosis congenita reveals the therapeutic functions of Wnt agonists	2016	49
	organoids	gene knockout intestinal organoids form tumors in mice kidney after subcapsule injection	2015	51
	organoids	organoids model reveals the function of APC and P53 in intestinal neoplasia	2015	52
	organoids	organoids model reveals the function of TGF-β in colorectal cancer (CRC) formation	2016	53
	organoids	combinatorial drug responses in organoids model (colorectal cancer [CRC])	2016	54
Gene correction therapy	HSCs	gene-corrected patient HSCs showed functional recovery in X-linked chronic granulomatous	2017	65
	iPSCs	gene-corrected patient iPSCs recovered β-globin (HBB) expression	2015	59
	organoids	gene-corrected patient organoids showed functional recovery in cystic fibrosis	2013	50
	HSCs	gene-corrected patient HSCs recovered β-globin (HBB) expression in β-thalassemia	2016	61
	iPSCs	gene-corrected patient iPSCs showed functional recovery in hemophilia A	2015	63
	iPSCs	gene-corrected patient iPSCs showed normal phenotypes in Huntington’s disease	2017	64
Anti-virus therapy	T cells	CXCR4-disrupted T cells showed HIV resistance	2015	69
	HSCs	CCR5-disrupted HSCs presented HIV resistance	2017	70
	iPSCs	CCR5-disrupted iPSCs and its derived blood cells showed HIV resistance	2015	71
	iPSCs	CCR5Δ32 iPSCs and its derived cells showed HIV resistance	2014	72
	iPSCs	CRISPR/Cas9-expressed iPSCs showed HIV resistance by elimination of virus RNA	2015	74
Anti-tumor therapy	CAR T cells	the anti-tumor efficacy of CAR T cells was enhanced through disrupting the PD-1 gene	2017	77
	CAR T cells	the anti-tumor efficacy of CAR T cells was improved by fusing CD19 CAR to the TRAC gene	2017	78
	iPSCs	NK cells derived from ADAM17-disrupted iPSCs presented higher HIV resistance	2016	79

hESCs, human embryonic stem cells.