Table 1 All-in-one rAAV vector-based CRISPR delivery.
From: Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery
Nuclease | Size (bp) | Gene | Administration route | Editing efficiency | rAAV serotype | Animal model | References |
|---|---|---|---|---|---|---|---|
CasMINI_3.1 | 1587 | Nr2e3 | Subretinal | >70% in GFP⁺ cells | 8 | RhoP23H/+ mouse model of RP | Cui et al. [22] |
Nme2-ABE8e | 3840 | Fah | Intravenous | 0.34% | 9 | Mouse model of HT1 | Zhang et al. [23] |
IscB-ABE | 2793 | Fah | Intravenous | 15% | 8 | Mouse model of HT1 | Guo et al. [24] |
IscB-CBE | 2136 | hDMD | Intramuscular | 7% | 9 | Humanized DMDE51del mice | Xiao et al. [25] |
TnpB | 1224 | Pcsk9 | Intravenous | 56% | 9 | C57BL/6 | Marquart et al. [26] |
