Sequence: LRKLRKRLLR
PMO (splice-switching morpholino)
| Experiment Id | EXP000798 |
|---|---|
| Paper | Amphiphilic lipopeptide significantly enhances uptake of charge-neutral splice switching morpholino |
| Peptide | Myr-ApoE (peptide amphiphile) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.125–0.5 µM (as lipopeptide–PMO conjugate) |
| Rna Concentration | 0.125–0.5 µM (PMO equivalent) |
| Mixing Ratio | |
| Formulation Format | covalent lipopeptide–PMO conjugate (self-assembled nanoparticles) |
| Formulation Components | Myr-ApoE-Ahx-Cys thioether-linked to 3'-maleimide PMO |
| Size Nm | 587.00 |
| 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 | ~4-fold lower concentration required vs ApoE-PMO to achieve similar splice-switching; ~30% higher activity vs ApoE-PMO |
| Output Units | |
| Output Notes | Myr-ApoE-PMO tested at 0.125, 0.25, 0.5 µM; compared against ApoE-PMO at 0.5–2 µM. |
| Toxicity Notes | No cytotoxicity at tested concentrations (MTS assay). |
| Curation Notes |