ANTP-ABE8e (covalent CPP fusion)
Sequence: RQIKIWFQNRRMKWKK
| Experiment Id | EXP001444 |
|---|---|
| Paper | Safer and efficient base editing and prime editing via ribonucleoproteins delivered through optimize |
| Peptide | ANTP-ABE8e (covalent CPP fusion) |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | no |
| Label Confidence | medium |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | Dose reported as ~4.5 µg ABE RNP per eye for CPP-fusion comparison experiment |
| Rna Concentration | Guide RNA present in RNP; delivered as assembled RNP |
| Mixing Ratio | Direct delivery of CPP-fused ABE8e RNP; formulations included sucrose as described for free RNP experiments |
| Formulation Format | CPP-fused ABE8e RNP (direct subretinal delivery, no LNP) |
| Formulation Components | CPP-fused ABE8e protein + sgRNA (rd12-A6) (+ sucrose/NaCl formulation as indicated) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | rd12 mouse model of Leber congenital amaurosis (Rpe65 nonsense mutation) |
| Administration Route | Subretinal injection (1 µl per eye) |
| Output Type | In vivo functional outcome: retinal function rescue (scotopic flash ERG b-wave) and on-target editing (sequencing of RPE DNA/cDNA) |
| Output Value | |
| Output Units | |
| Output Notes | CPP-fused ABE8e RNPs were evaluated in vivo; ERG rescue and low-percentage on-target editing were detected, but fused CPPs did not substantially improve outcomes compared with unmodified RNP under their conditions. |
| Toxicity Notes | |
| Curation Notes |