Table 2.

CRISPR/Cas-mediated correction of Dmd mutations in mice

Strategy	Mouse Model	CRISPR/Cas Type	DMD Mutations	sgRNA Targeting Site	Genome Editing Outcome	Reference Nos.
Exon deletion	mdx	SpCas9 and SaCas9	Ex23 point mutation	i22, i23	Δ Ex23	230, 289, 383
	mdx/Utr+/–	SpCas9 and SaCas9	Ex23 point mutation	i20, i23	Δ Ex21–23	104
	mdx	SpCas9	Ex23 point mutation	i20, i23	Δ Ex21–23	446
	mdx4cv	SpCas9 and SaCas9	Ex53 point mutation	i51, i53	Δ Ex52–53	29
Exon skipping	Δ Ex50	SpCas9	Δ Ex50	e51 SA	Skp. Ex51	7
Exon reframing	mdx	SpCas9	Ex23 point mutation	e23	Ex23 reframing	231
	Δ Ex50	SpCas9	Δ Ex50	e51 SA	Ex51 reframing	7
	mdx4cv	SpCas9	Ex53 point mutation	e53	Ex53 reframing	29
Exon HDR	mdx	SpCas9	Ex23 point mutation	e23	Ex23 HDR	231
	mdx	LbCpf1	Ex23 point mutation	e23	Ex23 HDR	469
	mdx4cv	SpCas9	Ex53 point mutation	e53	Ex53 HDR	29

i, intron; e, exon; Δ, deletion; SA, splice acceptor; Skp., skipping.