Table 1.
Allele-Specific Inactivation and/or Correction in Different Cell Types from Humans and Mice
| Cell Lines | sgRNA | % NHEJ | % Allele-Specific Inactivation following NHEJ | % HDR | % Allele-Specific Correction following HDR |
|---|---|---|---|---|---|
| HeLa | C21 pan allelic | 13.5 | NA | 9.3 | NA |
| Patient fibroblasts (R465W mutation) | C18 allele specific | 60 | 100 | 0 | NA |
| Dnm2R465W/+ mouse myoblasts | C19 allele specific | 95 | 100 | 0 | NA |
| C20 allele specific | 90.6 | 100 | 6.2 | 100 | |
| C22 pan allelic | 25 | NA | 0 | NA |
More than 55 cell clones were sequenced per sgRNA that were either pan allelic or allele specific. NA, not applicable, as there was no DNM2 mutation in HeLa cells or utilization of pan-allelic sgRNAs.