. Author manuscript; available in PMC: 2023 Feb 1.

Published in final edited form as: Adv Drug Deliv Rev. 2021 Dec 20;181:114087. doi: 10.1016/j.addr.2021.114087

Table 2.

Clinical applications of RNP-mediated genome editing.

Target cells	Editing methods	Clinical application	Reference
T cells	NHEJ	HIV infection treatment	[177]
T cells	NHEJ, HDR, base editing	CAR-T cell immunotherapy development	[66, 67, 178, 179]
B cells	HDR	Protein secreting plasma cells for protein deficiency treatment (ex. hemophilia B)	[69]
HSPCs	NHEJ, HDR, base editing	Hematologic disorder treatment (ex. sickle cell disease)	[41, 73, 74, 76, 180–183]
	NHEJ, HDR	Immunodeficiency disorder treatment (ex. X-linked severe combined immunodeficiency)	[184–187]
	HDR	Metabolic disorder treatment (ex. mucopolysaccharidosis type I)	[188]
iPSCs	HDR	Skin disorder treatment (ex. recessive dystrophic epidermolysis bullosa)	[77]
	HDR	Hematologic disorder treatment (ex. sickle cell disease)	[78]
	HDR	Lung disorder treatment (ex. cystic fibrosis)	[79]
Zygotes	NHEJ, HDR, base editing	Germline gene editing	[80–85, 88, 91]
Zygotes	HDR	Germline gene therapy (ex. hypertrophic cardiomyopathy)	[89, 90]
Retinal cells	NHEJ	Ocular disorder treatment (ex. age-related macular degeneration)	[100, 104]
Cochlear cells	NHEJ, base editing	Sensorineural hearing loss treatment	[101, 103]

HPSCs, hematopoietic stem and progenitor cells; iPSCs, induced pluripotent stem cells; NHEJ, non-homologous end joining; HDR, homology directed repair; HIV, human immunodeficiency virus; CAR, chimeric antigen receptor.