. 2020 Jan 22;21(3):733. doi: 10.3390/ijms21030733

Table 1.

Clustered regularly interspaced short palindromic repeats (CRISPR) studies on Duchenne muscular dystrophy (DMD) treatment using induced cardiomyocytes (iCMs) from human induced pluripotent stem cell (hiPSCs).

Model	Strategy, Nuclease	Delivery, Vector/s	Cardiac Findings				Reference
Model	Strategy, Nuclease	Delivery, Vector/s	RT-PCR	WB	ICC	Function, etc.	Reference
Δex46–51, Δex46–47, ex50dup. from patient fibroblasts	DMD Δex45–55, SpCas9	Nucleofection, dual plasmid	Skipping observed	DYS observed	DYS⁺ cells observed	iCMs from treated iPSCs (Δex46–51, Δex46–47) had reduced CK release in hypo-osmotic conditions	2016 Young et al. [92]
Δex48–50 from patient fibroblasts	DMD ex51 NHEJ repair or skipping, LbCpf1/AsCpf1	Nucleofection, single plasmid	Reframing, skipping observed	DYS observed in all strategies	DYS⁺ cells observed in all strategies	iCMs from reframed iPSCs had significantly more mitochondria and increased respiratory capacity	2017 Zhang et al. [94]
Δex8–9, CRISPR-generated from healthy PBMCs in study; Δex3–7 from patient (source not stated)	DMD Δex3-9, Δex6-9, Δex7–11, SpCas9	Nucleofection, dual plasmid	Skipping observed in all strategies	DYS observed in all strategies (Δex7–11 had least DYS)	DYS⁺ cells observed in all strategies	Ca²⁺ dynamics improved after treatment, but only significant in Δex3–9 iCMs; EHM from treated iCMs had enhanced contractility, with Δex3–9 showing best results	2017 Kyrychenko et al. [95]
Δex48–50, pseudo-ex47, ex55–59dup. from patient PBMCs	DMD ex51 skipping, cryptic splice site removal in ex47, Δ55–59dup., respectively, SpCas9	Nucleofection, single plasmid	Skipping observed in all strategies	DYS observed in all strategies	DYS⁺ cells observed in all strategies	EHMs from corrected iCMs had significantly improved contractile force; 30% or 50% DYS⁺ CMs sufficient for partial or complete recovery, respectively	2018 Long et al. [96]
Δex51 from patient PBMCs	DMD ex50 skipping, dSaCas9-TAM	Lipotransfection, single plasmid (with separate Ugi plasmid)	Skipping observed	DYS observed	DYS⁺ cells observed	iCMs from treated iPSCs had significantly reduced CK release in hypo-osmotic conditions	2018 Yuan et al. [97]
Δex48–50 from patient fibroblasts	DMD ex51 NHEJ repair or skipping, SpCas9	Nucleofection, single plasmid	-	67%–100% DYS of WT observed	DYS⁺ cells observed	-	2018 Amoasii et al. [98]
Δex44 from patient PBMCs	DMD ex43, 45 NHEJ repair or skipping, SpCas9	Nucleofection, single plasmid	-	DYS observed	DYS⁺ cells observed	-	2019 Min et al. [99]

Abbreviations: RT-PCR, reverse transcription-polymerase chain reaction; WB, Western blot; ICC, immunocytochemistry; DYS, dystrophin; CK, creatine kinase; NHEJ, non-homologous end-joining; PBMCs, peripheral blood mononuclear cells; EHM, engineered heart muscle.