Sequence: LRKLRKRLLR
PMO (splice-switching morpholino)
| Experiment Id | EXP000797 |
|---|---|
| Paper | Amphiphilic lipopeptide significantly enhances uptake of charge-neutral splice switching morpholino |
| Peptide | ApoE (CPP fragment) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.5–2 µM (as peptide–PMO conjugate) |
| Rna Concentration | 0.5–2 µM (PMO equivalent) |
| Mixing Ratio | |
| Formulation Format | covalent peptide–PMO conjugate (self-assembled nanoparticles) |
| Formulation Components | Ac-ApoE-Ahx-Cys thioether-linked to 3'-maleimide PMO |
| Size Nm | 261.00 |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | SMA Type I patient-derived fibroblasts (GM03813) |
| Animal Model | |
| Administration Route | |
| Output Type | splice-switching activity (RT-PCR; % full-length SMN2 transcript) |
| Output Value | Increased exon-7 inclusion vs PMO alone (dose-dependent) |
| Output Units | |
| Output Notes | ApoE-PMO tested at 0.5, 1, 2 µM; RT-PCR quantification of Δ7SMN2 → full-length SMN2 |
| Toxicity Notes | No cytotoxicity at tested concentrations (MTS assay). |
| Curation Notes |