The Development and Application of a Base Editor in Biomedicine

Table 2

Summary of application of base editor in gene therapy.

First author	Publication years	Species (tissues)	Category of base editor	Delivery	Disease	Gene	Mutation efficiency	Off-target
Alexis C. Komor [15]	2016	Mouse astrocytes	BE3	Plasmids	Alzheimer’s disease	APOE4	58-75%	4.6–6.1% indels at the targeted locus
Alexis C. Komor [15]	2016	Human cell	BE3	Plasmids	Breast cancer	P53	3.3-7.6%	≤0.7% indel formation
Puping Liang [50]	2017	Human embryos	YEE-BE3	Injecting mRNA and gRNA	β-Thalassemia	HBB	22.9%	No off-target in top 10 predicted sites
Alexandra C. Chadwick [82]	2017	Adult mice	BE3	Adenoviral vectors	High blood cholesterol	PCSK9	Average 24%	≤1% indel formation
Seuk-Min Ryu [70]	2018	Adult mice	ABE	Adeno-associated virus	Duchenne muscular dystrophy	DMD		No off-target mutations at homologous sites with up to three mismatches
Luke W Koblan [20]	2018	Human fibroblasts	BE4, BE4max–P2A–GFP, AncBE4max–P2A–GFP	Plasmids	Glycosylation type 1f	MPDU1	32-77%	N.R.
Lukas Villiger [76]	2018	Adult mice	nSaKKH-BE3	Adeno-associated virus	Autosomal recessive liver disease phenylketonuria	PAH	Average 25.1%	No C∙ G to T∙ A conversions or indel formations in ten potential off-target loci
			dLbRR-BE
Alexandra C. Chadwick [83]	2018	Adult mice	BE3	Adenoviral vectors	Hyperlipidemic	ANGPTL3	35%	No off-target in top 10 predicted sites
Yanting Zeng [84]	2018	Human embryos	BE3	Injecting mRNA and gRNA	Marfan syndrome	FBNA	89%	No off-target and indels were detected in 32 potential off-target sites
Maria Paz Zafra [85]		Mouse intestinal organoids	FNLS-BE3	Plasmids	Colorectal cancers19	Apc	>97%	Less than 1% indels
Xinjian Li [54]	2020	Human U87 and U251 cells	CjABE	Adeno-associated virus	Glioblastoma	TERT	70-80%	N.R.
Xiang Lin [56]	2020	Human SMA motor neurons	SaCas9n-KKH-ABE	Injecting mRNA and gRNA	Spinal muscular atrophy	SMN2	5-40%	No detectable off-target effects in DNA level
Colin K.W.Lim [78]	2020	Adult mice	BE3	Adeno-associated virus	Amyotrophic lateral sclerosis	SOD1	N.R.	N.R.
Chun Qing Song [86]	2020	Adult mice	ABE6.3	Plasmids	Tyrosinemia	FAH		One off-target site
Jonathan M. Levy [87]	2020	Adult mice	BE3	Adeno-associated virus	Niemann-pick disease	Npc1	10-80%	One off-target site