Table 2.

Gene therapy strategies and therapeutic effects for major CNS diseases.

Disease	Target Gene (Protein)	Mechanism	Reference
PD	AADC	Replace	[16]
	GAD	Replace	[17]
	GBA1	Replace	[33]
	SNCA	Silence	[34]
AD	APOE4	Silence/immunotherapy	[20]
	APOE2	Replace	[35]
	APP (amyloid-beta)	Silence	[22]
	MAPT (tau)	Silence/immunotherapy	[36]
	PQBP1	Replace	[23]
ALS	SOD1	Silence	[37]
	C9orf72	Silence	[38]
	TARDBP	Silence	[39]
SMA	SMN1	Replace	[24]