Table 4.

Gene editing therapy for FH in development

Target	Delivery Strategy	Model	Editing tools	Editing efficiency	Lipid-Lowering Effect	References
PCSK9	LNP	NHPsa	ABE8.8	∼79.4%	60%	Musunuru et al.134
	LNP	Mice	ABE	61%	58%	Rothgangl et al.133
	LNP	NHPsa	ABE	26%	14%	Rothgangl et al.133
	ADV	Mice	CRISPR-Cas9	>50%	35-40%	Ding et al.132
ANGPTL3	ADV	LDLR−/− Mice	CBE3	∼35% (ANGPTL3)	51%	Chadwick et al.135
	LNP	Mice	CRISPR-Cas9	38.5%	56.8%	Qiu et al.136
	AAV9	Mice	ABE4max	63.30%	61%	Zuo et al.137
	GalNAc-LNP	Mice	ABE8.8	64%	/	Lee et al.138
	GalNAc-LNP	NHPs1	ABE8.8	∼61%	35%	Kasiewicz et al.139

^aNHPs: non-human primates.