Splice site editing |
Human hepatocytes in vitro |
NA |
>60% splice site editing achieved at splice site |
PCSK9 expression |
Human hepatocytes in vitro |
NA |
PCSK9 expression reduced by 55% |
Splice site editing |
Mouse in vivo |
4–5 |
A 70% liver base editing of the splice site |
Splice site editing |
Cynomolgus monkeys in vivo |
2–3 |
A 63% base-editing frequency of the PCSK9 splice-site after two weeks |
PCSK9 expression |
Cynomolgus monkeys in vivo |
2–3 |
A 81% reduction in blood PCSK9 |
LDL-C |
Cynomolgus monkeys in vivo |
2–3 |
A 65% reduction in blood LDL-C |