Sequence: VPA–XLRKLRKRLLRXC-NH2 (as shown in paper schematic)
splice-switching PMO (morpholino)
| Experiment Id | EXP001489 |
|---|---|
| Paper | Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outco |
| Peptide | (N)-VPA-ApoE |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | no |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.5–1 µM (P-PMO) (toxicity tested up to 4 µM) |
| Rna Concentration | 0.5–1 µM (PMO, covalent to peptide) |
| Mixing Ratio | 1:1 (covalent conjugate) |
| Formulation Format | covalent VPA–CPP–PMO conjugate |
| Formulation Components | (N)-VPA-ApoE-PMO (amide-linked VPA) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | SMA Type I patient-derived fibroblasts (GM03813, Coriell) |
| Animal Model | |
| Administration Route | |
| Output Type | SMN2 splice switching (RT-qPCR) |
| Output Value | Slight increase in FL-SMN2 (not statistically significant vs untreated) |
| Output Units | |
| Output Notes | (N)-VPA-ApoE-PMO showed poor efficacy vs VPA + ApoE-PMO co-treatment; splice correction not clearly demonstrated beyond noise. |
| Toxicity Notes | At 16 h, 4 µM (N)-VPA-ApoE-PMO showed significant cytotoxicity (reported cell viability ~72%). |
| Curation Notes |