Gene editing for corneal disease management

Table 2 Potential applications of zinc finger nucleases, transcription activator-like effector nucleases and Clustered Regularly Interspaced Short Palindromic Repeat/Clustered Regularly Interspaced Short Palindromic Repeat Associated Systems to develop novel disease models and innovative therapeutic strategies

Target gene	Target cell	ZFN/TALEN/CRISPR	Disease	Ref.
α-Globin	Human iPS	ZFN	α-thalassemia	[104]
Tnfrsf9	NOD mouse embryo	ZFN	Diabetes	[105]
HBV	Huh7 cells	ZFN	Hepatitis B	[106]
CCR5, CXCR4	CD4+ T cells	ZFN	HIV	[107]
CCR5, IL2RG	Multiple	ZFN	HIV, X-SCID	[108]
TCRα, β	T cells	ZFN	Leukemia	[109]
HBB	Human iPS cells	ZFN	Sickle cell anemia	[110]
PIG-A	Human ES, iPS cells	ZFN	PNH	[111]
gp91(phox)	Human iPS cells	ZFN	X-CGD	[112]
Albumin	Mouse hepatocytes	ZFN	Hemophilia A and B	[113]
SCN1A	Human iPS	TALEN	Epilepsy	[114]
PSIP1	HT1080, 293T, Jurkat	TALEN	HIV	[115]
HBB	Human iPS cells	ZFN/TALEN/CRISPR	Sickle cell anemia	[116]
gp91(phox)	Human iPS cells	TALEN	X-CGD	[117]
Ctnnb1, Apc	H2.35	TALEN	Hepatocellular carcinoma	[118]
hFVIII	Human iPS cells	TALEN	Hemophilia A	[119]
PLN R14del	Human iPS cells	TALEN	Cardiomyopathy	[120]
BUB1B	HCT116	TALEN	PCS (MVA)	[121]
MECP2	Monkey zygotes	TALEN	Rett syndrome	[122]
Sry, Uty	Mouse blastocysts	TALEN	NA	[123]
Dystrophin	Myoblasts	CRISPR/Cas9	DMD	[124]
FANCC	Patient fibroblasts	CRISPR/Cas9	Fanconi anemia	[125]
APC, SMAD4, TP53, KRAS, PIK3CA	Human intestinal epithelial organoids	CRISPR/Cas9	Colorectal cancer	[126]
FAH	Mouse liver	CRISPR/Cas9	Tyrosinemia	[127]
PTEN, TP53	Mouse liver	CRISPR/Cas9	Liver cancer	[128]
DMD	Mdx mouse zygotes	CRISPR/Cas9	DMD	[129]
B2M, CCR5	CD4+ T and CD34+ HSC	CRISPR/Cas9	NA	[130]
CFTR	CF intestinal organoids	CRISPR/Cas9	Cystic Fibrosis	[131]
C. parvum	HCT8	CRISPR/Cas9	Cryptosporidiosis	[132]
HCV	Huh7.5	FnCas9	Hepatitis C	[133]

ZFN: Zinc finger nuclease; TALEN: Transcription activator-like effector nucleases; CRISPR: Clustered Regularly Interspaced Short Palindromic Repeats; Cas9: CRISPR associated protein 9; HIV: Human immunodeficiency virus; NOD: Non-obese diabetic.