Table 1. Generation and treatment of animal models of human diseases using genome editing methods.

Species	Target gene	Disease	Technique	Editing Method		Reference
Generatiof disease animal models
Mouse	Fah	Tyrosinemia	Microinjection	SpCas9	mRNA	Li et al. (2015)
	Rag1, IL2RgammaC	Immunodeficient
	Notch3	Lateral meningocele syndrome	Microinjection	SpCas9	mRNA	Canalis et al. (2018)
	ATP6V1H	Osteoporosis	Microinjection	SpCas9	mRNA	Duan et al. (2016)
	Bril	Osteogenesis imperfecta (OI)	Microinjection	SpCas9	mRNA	Rauch et al. (2018)
	Sox9	Acampomelic campomelic dysplasia (ACD), Campomelic dysplasia (CD)	Microinjection	SpCas9	mRNA	Mochizuki et al. (2018)
	Dystropin	Duchenne Muscular Dystrophy (DMD)	Electroporation	Cytidine Base editor 3 (BE3)	RNP	Kim et al. (2017)
	p53, Lkb1, KRAS	Cancer	Intratracheal injection	SpCas9	AAV	Platt et al. (2014)
Rat	Tyrosine hydroxylase (TH)	Parikinson’s disease (PD)	Intracranial injection	SpCas9	AAV	Back et al. (2019)
pig	Huntingtin (HTT)	Huntington’s disease (HD)	Somatic cell nuclear transfer	SpCas9	plasmid	Yan et al. (2018)
	Parkin, Pink1	Parikinson’s disease (PD)	Somatic cell nuclear transfer	SpCas9	plasmid	Zhou et al. (2015)
Monkey	Dystropin	Duchenne Muscular Dystrophy (DMD)	Microinjection	SpCas9	mRNA	Chen et al. (2015)
Dog	Myostatin	Muscle hypertrophy	Microinjection	SpCas9	mRNA	Zou et al. (2015)
Rabbit	Myostatin	Muscle hypertrophy	Microinjection	SpCas9	mRNA	Lv et al. (2016)
	Dystropin	Duchenne Muscular Dystrophy (DMD)	Microinjection	SpCas9	mRNA	Sui et al. (2018)
	PAX4	Diabetes mellitus (DM)	Microinjection	SpCas9	mRNA	Xu et al. (2018)
Treatment of disease animal models
Mouse	F8	Hemophilia A	Patient-Derived iPSCs correction and transplantion	SpCas9	Plasmid	Park et al. (2015)
	F9	Hemophilia B	Intravenous injection	SaCas9	AAV	Ohmori et al. (2017)
	Dystropin	Duchenne Muscular Dystrophy (DMD)	Intramuscular injection,	SpCas9	AAV	Tabebordbar et al. (2016)
			Intraperitoneal injection,	SaCas9	AAV	Nelson et al. (2016)
			Intravenous injection,	SpCas9	AAV	Long et al. (2016)
			Retro-orbital injection	SpCas9	AAV	Bengtsson et al. (2017)
			Intramuscular injection	SpCas9	RNP with gold nanoparticle	Li et al. (2015)
			Intramuscular injection	Adenine Base Editor (ABE)	AAV	Ryu et al. (2018)
	SOD1	Amyotrophic lateral sclerosis (ALS)	Intravenous injection	SaCas9	AAV	Gaj et al. (2017)
	Fah	Tyrosinemia	Intravenous injection(Hydrodynamic injection)	SpCas9	Plasmid	Yin et al. (2014)
			Intravenous injection	SpCas9	AAV	Yin et al. (2016)
			Intravenous injection (Hydrodynamic injection)	Adenine Base Editor (ABE)	Plasmid	Song et al. (2019)
	LTR, Gag, Pol	HIV-1/AIDS	Intravenous injection,	SaCas9	AAV	Kaminski et al. (2016)
			Intravaginal injection,			Yin et al. (2017)
			Retro-orbital injection
	Huntingtin (HTT)	Huntington’s disease (HD)	Stereitactic injection	SpCas9	AAV	Monteys et al. (2017)
	Vegfr2	Age-related macular degeneration (AMD)	Intravitreal injection	SpCas9	AAV	Huang et al. (2017)
	Vegfr		Subretinal injection	SpCas9	RNP with liposome	Kim et al. (2017)
	Vegfa		Intravitreal injection	CjCas9	AAV	Kim et al. (2017)
	Hif1a		Intravitreal injection	LbCpf1	AAV	Koo et al. (2018)
	Bace1	Alzheimer’s disease (AD)	Intraacranial injection	SpCas9	RNP with peptide	Park et al. (2019)
Rat	Rho	Retinal dystropy	Subretinal injection	SpCas9	plasmid	Bakondi et al. (2016)
Dog	Dystropin	Duchenne Muscular Dystrophy (DMD)	Intramuscular injection, Intravenous injection	SpCas9	AAV	Amoasii et al. (2018)
Pig	Alb	Liver failure, traumatic shock	Microinjection	SpCas9	mRNA	Peng et al. (2015)