Sequence: Ac-AAAA-X-LRKLRKRLLR
| Experiment Id | EXP000259 |
|---|---|
| Paper | Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and C |
| Peptide | A4-ApoE(141–150) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | medium |
| In Vitro Functional Effect | no |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.5–5 µM |
| Rna Concentration | Equimolar |
| Mixing Ratio | 1:1 covalent conjugate |
| Formulation Format | Peptide–PMO conjugate |
| Formulation Components | A4-ApoE(141–150)-PMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | SMA patient-derived fibroblasts (GM03813) |
| Animal Model | |
| Administration Route | |
| Output Type | SMN2 exon-7 inclusion (RT-qPCR) |
| Output Value | Measured |
| Output Units | |
| Output Notes | In vitro screening |
| Toxicity Notes | No toxicity ≤5 µM |
| Curation Notes |