Table 1.

Summary of recent advancements of in vivo pre-clinical models as a therapeutic approach.

Strategies	Disease	Model	Reference
ZFN	Hemophilia A and B	Mouse	Li et al., 2011 [26] Sharma et al., 2015 [27] Anguela et al., 2013 [28] Park et al., 2016 [29]
	Human immunodeficiency Virus (HIV-1)	Human	Holt et al., 2010 [30]
	Mucopolysaccharidosis II (MPS II) or Hunter’s syndrome	Mouse	Laoharawee et al., 2016 [31] Laoharawee et al., 2018 [32] Sawamoto et al., 2018 [33]
Transcription activator-like effector nucleases (TALENS)	Human immunodeficiency Virus (HIV-1)	Humanized Mouse	Benjamin et al., 2016 [34]
	Hepatitis B Virus (HBV)	Mouse	Weber et al., 2013 [35]
CRISPR-Cas9	Hereditary Tyrosinemia Type I (HTI)	Mouse	Yin et al., 2014 [36] Yin et al., 2016 [37] Pankowicz et al., 2016 [38]
	Human immunodeficiency Virus (HIV-1)	Human	Ebina et al., 2013 [27] Dai et al., 2016 [39]
	Human immunodeficiency Virus (HIV-1)	Primary CD4+ T cells	Liu et al., 2017 [40]
	Human immunodeficiency Virus (HIV-1)	Humanized Mouse	Yin et al., 2017 [41] Kaminski et al., 2016 [42]
	Age-related Macular Degeneration (AMD)	Mouse	Kim et al., 2017 [43] Kim et al., 2017 [44]
	Retinal Dystrophy	Rat; Ex vivo	Yanki et al., 2017 [10]
	Retinitis Pigmentosa	Mouse	Latella et al., 2016 [45] Wu et al., 2016 [46] Zhu et al., 2017 [47]
	Duchenne Muscular Dystrophy (DMD)	Mouse	Tabebordbar et al., 2016 [48] Long et al., 2016 [49] Nelson et al., 2016 [50] Bengtsson et al., 2017 [51]
	Amyotrophic Lateral Sclerosis (ALS)	Mouse	Gaj et al., 2017 [52]
	Haemophilia B	Mouse	Singh et al., 2018 [53]
	Rett syndrome	Mouse	Swiech et al., 2015 [54]
	Ornithine Transcarbamylase (OTC) deficiency	Mice	Yang et al., 2016 [55]
	Alpha-1 antitrypsin (AAT) deficiency	Mouse	Song et al., 2018 [56]
	Cancer	PD-L1+ Tumor xenograft model (in vivo); Human	Rupp et al., 2017 [57] Castillo et al., 2016 [58]
	Leber’s congenital amaurosis (LCA)	Human	Bainbridge et al., 2008 [59] Maguire et al., 2008 [60] Ruan et al., 2017 [61]