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.