Table 1.

Applications of the CRISPR/Cas9 system in nervous system diseases.

Pathology	Target gene	Model	Result	Reference
AD	Bace1 gene	5XFAD mouse, APP knock-in (KI) mouse	APP↓, Aβ↓	68
	APP gene	Mouse	Aβ↓	69
	APOE4 gene	Mouse	APOE4 allele→APOE3 allele	56
		iPSCs	Tau hyperphosphorylation↓	73,74
	ATP6V1 A gene	iPSCs	Neuronal activity↓	79
PD	Vps35 D620N	Mouse	Mouse model of PD	87
	α-synuclein gene	Pig	Pig model of PD	88
	Not mentioned	Monkey	Monkey model of PD	90
	DNAJC6	ESCs	Stem cell model of PD	91,92
	Not mentioned	Human pluripotent stem cells	Human brain-like organoids model of PD	93
	SNCA gene	Mouse	SNCA↓, α-synuclein↓	95,96
	VPS59 D620N	Cell	Mitosis↓	99
HD	HHT gene	Pig	Pig model of HD	107
	HHT gene	HD140Q-Ki mice	Early neuropathological changes in the striatum↓	109
GBM	TLE4, IKZF2, EIF5A, TMEM184B	CAR-T cell	Death of CAR-T cells↓	116
	relA/p65, NPLOC4	GBM cell	Death of GBM cells↑	116
	Gene of IFNγR signaling pathway	GBM cell	Drug resistance↑	117
Epilepsy	KCNA1 gene	Mouse	Kv1.1↑, frequency of seizures↓	124
	KCNB1 gene	Mouse	Kv2.1↑, abnormal EEG activity↑	127
	SCN1A gene	Mouse	Nav1.1↑, epileptic symptoms↓	131
ALS	C9orf72	Mouse	Synaptic dysfunction↓	143
	SOD1 gene	SOD1G93A mutant neonatal mice	Quantity of motor neurons↑ Muscle strength↑ Survival rate↑	147,148