TABLE 2.
Characteristics of the Cas9 used in the different therapeutic approaches for FSHD.
| Abbreviation | Species | PAM motif | Packaging size | Strategy | Reference |
|---|---|---|---|---|---|
| SpCas9 | Streptococcus pyogenes | 5’NGG-3′ | ∼ 4.2 kb | Double strand break | Himeda et al (2021); Joubert et al (2020); Goossens et al (2019) |
| KRAB | Himeda et al (2021); Das et al (2021) | ||||
| ABE | Sikrova et al. (2021) | ||||
| SaCas9 | Staphylococcus aureus | 5’-NNGRRT-3’. | ∼ 3.2 kb | ABE | Sikrova et al (2021) |
| dSaCas9 | Staphylococcus aureus | 5′-NNGRRT-3′ | ∼ 3.2 kb | Epigenetic regulator | Himeda et al (2016) |
| CjCas9 | Compylobocter jejuni | 5′-NNNVRYM-3′ | ∼ 2.9 kb | ABE | Sikrova et al (2021) |
| NmCas9 | Neisseria meningitidis | 5′-NNNNGATT-3′ | ∼ 3.5 kb | Double strand break | Joubert et al (2020) |
Packaging site include Cas9 and sgRNA together.
N=A/G/C/T; R=G/A; V=G/C/A; Y=C/T; M=A/C.
ABE, adenine base editor.