Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy

Table 2

Summary of papers utilizing various CRISPR-Cas9 strategies to target DMD mutations in patient-derived cells.

Strategy	Target cell	DMD type	Genome editing target	Deletion size	Ref.
Mono or multiexon deletion	Myoblast	ΔEx48–50	Ex51; Ex45–55	336 kbp	[50]
Multiexon deletion	iPSC	ΔEx46–51; ΔEx46-47; Dup ex50	Ex45–55	530 kbp; 670 kbp; 725 kbp	[12]
Exon deletion	Myoblast	ΔEx48–50; ΔEx45–52	Ex51; Ex44–54; Ex53		[49]
Exon deletion	mdx mice (in vivo)	Nonsense mut in Ex23	Ex23	~0.5 kbp	[52]
Exon deletion	mdx mice (in vivo)	Nonsense mut in Ex23	Ex23	~1.2 kbp	[51]
Exon deletion	mdx mice (in vivo)	Nonsense mut in Ex23	Ex23	~0.3 kbp	[53]
Exon skipping	iPSC	ΔEx44	Ex45	18 bp	[13]
Exon skipping	Myoblast	ΔEx48–50; ΔEx45–52	Ex51; Ex53		[49]
Frame shifting	iPSC	ΔEx44	Ex45	2 bp insertion	[13]
Frame shifting	Myoblast	ΔEx48–50; ΔEx45–52	Ex51; Ex53		[49]
Frame shifting and exon deletion	Myoblast	ΔEx51–53	Ex50; Ex54	>160 kbp	[57]
Exon knock-in	iPSC	ΔEx44	Ex45		[13]
Exon knock-in and exon deletion	mdx mice (in vivo)	Nonsense mut in Ex53	Ex52-53; Ex53		[55]