Sequence: Ac-(HS)XLRKLRKRLLRXC-NH2 (as shown in paper schematic)
splice-switching PMO (morpholino)
| Experiment Id | EXP001490 |
|---|---|
| Paper | Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outco |
| Peptide | (O)-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 | (O)-VPA-ApoE-PMO (ester-linked VPA, hydrolysable) |
| 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 | (O)-VPA-ApoE-PMO did not improve splice switching vs ApoE-PMO alone; functional benefit not clearly demonstrated. |
| Toxicity Notes | At 4 h, 4 µM (O)-VPA-ApoE-PMO caused a slight decrease in viability; higher toxicity (~80% viability) noted in some 16 h conditions compared with untreated. |
| Curation Notes |