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. 2022 Sep 22;11(19):2964. doi: 10.3390/cells11192964

Table 2.

CRISPR-mediated therapeutic strategies in human DMD cells.

Strategy Mutation Nuclease Target Region DMD Model Delivery Reference
Double-cut
exon exicision		 ΔEx46-51 SpCas9 i44, i55 human iPSCs electroporation [38]
ΔEx52 SpCas9 i50, i51 human iPSCs AAV6 [39]
ΔEx49-50,
ΔEx50-52,
ΔEx51-53,
ΔEx51-56		 SaCas9 Ex47, Ex58 human myoblasts lentivirus [40]
Dup Ex18-30 SpCas9 i27 human myoblasts lentivirus [122]
Dup Ex55-59 SpCas9 i54 human iPSCs nucleofection [150]
Dup Ex2 SpCas9 i2 human myoblasts lentivirus [151]
Dup Ex3-16 SpCas9 i9 human myoblasts lentivirus [152]
Dup Ex18-25 SpCas9 i25 human myoblasts lentivirus [153]
ΔEx48-50 SpCas9 i50, i51 human myoblasts electroporation [154]
ΔEx48-50 SpCas9 i44, i55 human myoblasts electroporation [154]
ΔEx51 SpCas9,
AsCas12a		 i44, i55;
i45, i54		 human myoblasts electroporation [155]
ΔEx45-52 SpCas9 i52, i53 human myoblasts adenovirus [156,157]
ΔEx45-52,
ΔEx48-50		 SpCas9,
eSpCas9(1.1)		 i43, i54 human myoblasts adenovirus [156,157,158]
ΔEx8-9 SpCas9 i2, i7;
i5, i7;
i6, i11		 human iPSCs nucleofection [159]
ΔEx3-7 SpCas9 i7, i9 human iPSCs nucleofection [159]
Ex23 mut SpCas9 i22, i23 mouse myoblasts lipotransfection [160]
Single-cut
exon skipping
and reframing		 ΔEx44 SpCas9 Ex45 human iPSCs nucleofection [132]
ΔEx48-50 SaCas9-KKH Ex51 human iPSCs nucleofection [133]
ΔEx43,
ΔEx45		 SpCas9 Ex44 human iPSCs nucleofection [134]
ΔEx52 SpCas9 Ex51 human iPSCs nucleofection [134]
ΔEx48-50 SpCas9-VRQR Ex51 human iPSCs nucleofection [135]
ΔEx48-50 LbCas12a,
AsCas12a		 Ex51 human iPSCs nucleofection [142]
ΔEx48-50 SpCas9 Ex51 human iPSCs nucleofection [150]
Pseudo Ex47 SpCas9 i47 human iPSCs nucleofection [150]
ΔEx48-50 eSpCas9(1.1) Ex51 human myoblasts adenovirus [158]
HDR-based
knockin		 i6 mut SpCas9 i6 canine myoblasts nucleofection [143]
ΔEx44 SpCas9 Ex44 human iPSCs electroporation [161]
Base editing ΔEx51 ABEmax-nSpCas9 Ex50 human iPSCs nucleofection [146]
ΔEx51 CBE-nSaCas9 Ex50 human iPSCs lipotransfection [162]
Prime editing ΔEx51 PE2-dSpCas9 Ex52 human iPSCs nucleofection [146]
Ex6 mut PE2-nSpCas9 Ex6 human myoblasts electroporation [163]

Δ, deletion; Ex, exon; i, intron; iPSC, induced pluripotent stem cell; AAV, adeno-associated viral vector; Dup, duplication; mut, mutation.