Table 2 Protein trans-splicing rAAV vectors encoding split-CRISPR molecules.
From: Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery
Nuclease | Gene | Administration route | Editing efficiency | rAAV serotype | Animal model | References |
|---|---|---|---|---|---|---|
CBE4 | SOD1 G93A | Intrathecal | 1.2% | 9 | mouse model of ALS | Lim et al. [28] |
CBE3.9max | Npc1 I1061T | Retro-orbital | 0.3% (unsorted), 42% (sorted) | 9 | Npc1I1061TÂ homozygous mice | Levy et al. [29] |
ABEmax | Rpe65 R44X | Subretinal | 13.5% | 9 | rd12 mouse model of LCA | Jo et al. [30] |
v3emPE | Pcsk9 Q155H | Retro-orbital | 39% | 9 | C57BL/6 | Davis et al. [31] |
PEmax | Pde6a D670G | Subretinal | 9.4% | 2.NN | Pde6anmf363/nmf363 mice | Liu et al. [32] |
PESpRY | Pde6b R560C | Subretinal | 40.9% (unsorted), 76.3% (sorted) | 8 | Pde6brd10Â mice | Qin et al. [33] |
