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

Table 1

Reported DMD-iPSC lines and genotypes.

Patient information (sex/age/cell type)	iPSC reprogramming method	Mutation description	Ref.
Male/6 YR/fibroblast (Coriell ID: GM04981)	Multiple lentiviral vectors	ΔExons 45–53	[8]
Male/28 YR/fibroblast (Coriell ID: GM05089)	Multiple lentiviral vectors	ΔExons 3–5	[8]
Male/9 YR/fibroblast (Coriell ID: GM05169)	Multiple retroviral vectors	ΔExons 4–43	[11]
Male/3 YR/fibroblast	Multiple retroviral vectors	ΔExon 44	[10]
Male/9 YR/fibroblast	Multiple retroviral vectors	ΔExons 46-47	[10]
Male/9 YR/fibroblast (Coriell ID: GM05169)	Multiple sendai virus vectors	ΔExons 4–43	[9]
Male/10 YR/fibroblast (Coriell ID: GM03783)	Multiple sendai virus vectors	ΔExons 3–17	[9]
Male/23 YR/fibroblast (Coriell ID: GM04327)	Multiple sendai virus vectors	Exons 5–7 duplication	[9]
Male/18 YR/fibroblast (Coriell ID: GM05127)	Multiple sendai virus vectors	DNA 5533 G→T (protein E→X)	[9]
Male/11 YR/fibroblast (Coriell ID: GM03781)	Multiple sendai virus vectors	ΔExons 3–17	[9]
Male/NA/fibroblast	Polycistronic lentivirus vector	ΔExons 46–51	[12]
Male/NA/fibroblast	Polycistronic lentivirus vector	ΔExons 46-47	[12]
Male/NA/fibroblast	Polycistronic lentivirus vector	Exon 50 duplication	[12]
Male/3 YR/fibroblast	Multiple episomal vectors	ΔExon 44	[13]
Male/31 YR/T lymphocytes	Multiple sendai virus vectors	ΔExons 48–54	[14]
Male/13 YR/T lymphocytes	Multiple sendai virus vectors	ΔExons 46-47	[14]
Male/18 YR/fibroblast	Multiple lentiviral vectors	ΔExons 48–50	[15]
Male/14 YR/fibroblast	Multiple lentiviral vectors	ΔExons 47–50	[15]
Male/13 YR/fibroblast	Multiple lentiviral vectors	DNA 3217 G→C (protein E→X)	[15]
Male/10 YR/fibroblast	Multiple lentiviral vectors	ΔExons 45–52	[15]
Male/10 YR/fibroblast	Multiple lentiviral vectors	DNA 10171 C→T (protein R→X)	[15]
Male/8 YR/fibroblast	Multiple lentiviral vectors	DNA 4918-4919 ΔTG	[15]
Male/20 YR/fibroblast	Multiple lentiviral vectors	DNA 7437 G→A (protein W→X)	[15]

NA: not available; YR: years old.