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. 2021 Apr 3;29(11):3179–3191. doi: 10.1016/j.ymthe.2021.04.002

Table 2.

Summary of animal models of DMD generated by the CRISPR-Cas system

Subject Animal Strategy Nuclease DMD mutation Target gene region(s) Strain Delivery Reference
Disease modeling mouse exon deletion SpCas9 DMD exon 45 deletion DMD intron 44 and 45 C57BL/10 and DBA/2 microinjection 81
SpCas9 Dmd exon 50 deletion Dmd intron 49 and 50 C57BL/6J not indicated 49
SpCas9 Dmd exon 44 deletion Dmd intron 43 and 44 C57BL/6J microinjection 47
SpCas9 Dmd exon 8–34 deletion Dmd intron 7 and 34 C57BL/6J xCBA microinjection 83
SpCas9 Dmd exon 50 deletion Dmd intron 49 and 50 not indicated not indicated 84
SpCas9 Dmd exon 43, 45, and 52 deletion Dmd intron 42 and 43
Dmd intron 44 and 45
Dmd intron 51 and 52
C57BL/6 microinjection 85
frameshift mutation CjCas9 Dmd frameshift mutation in exon 23 Dmd exon 23 C57BL/6J microinjection 40
base editing BE3 Dmd nonsense mutation in exon 20 Dmd exon 20 C57BL/6J and ICR mice microinjection and electroporation 82
rat exon deletion SpCas9 Dmd exon 3–16 deletions Dmd exon 3 and exon 16 Wistar-Imamichi rats microinjection 86
rabbit frameshift mutation SpCas9 DMD frameshift mutation in exon 51 DMD exon 51 New Zealand rabbits microinjection 87
pig base editing BE3, hA3A-BE3 DMD nonsense mutation in exon 13 DMD exon 13 Bama miniature pigs and large white pigs microinjection 88
monkey frameshift mutation SpCas9 DMD frameshift mutation in exon 4 and/or exon 46 DMD exon 4 and/or exon 46 Macaca mulatta rhesus monkeys microinjection 89

BE3, base editor 3; hA3A-BE3, hAPOBEC3A base editor 3; hDMD, humanized DMD.