Sequence: Ac-LRKLRKRLLRXC-NH2 (X = unspecified spacer as shown in paper schematic)
splice-switching PMO (morpholino)
| Experiment Id | EXP001491 |
|---|---|
| Paper | Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outco |
| Peptide | ApoE (co-treated with VPA) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | ApoE-PMO 0.5–1 µM; VPA 1–10 mM (co-treatment) |
| Rna Concentration | ApoE-PMO 0.5–1 µM |
| Mixing Ratio | N/A (co-administration; not covalent) |
| Formulation Format | co-treatment (small molecule + CPP–PMO) |
| Formulation Components | Valproic acid (VPA) + ApoE-PMO |
| 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) and SMN protein (western blot) |
| Output Value | Enhanced exon 7 inclusion vs either agent alone (e.g., FL-SMN2 up to ~1.89-fold at 4 h; ~2-fold at 16 h; SMN protein up to ~2.2-fold) |
| Output Units | |
| Output Notes | Co-treatment improved splice switching: 10 mM VPA + 1 µM ApoE-PMO (4 h) gave the only statistically significant FL-SMN2 increase reported for 4 h (~1.89-fold). Over 16 h, 2 mM VPA + ApoE-PMO gave ~2-fold FL-SMN2 increase and increased SMN protein (max reported ~2.2-fold with 2 mM VPA + 0.5 µM ApoE-PMO). |
| Toxicity Notes | Co-treatment generally well tolerated; 5 mM VPA + ApoE-PMO over 16 h reduced viability to ~82% (reported). |
| Curation Notes |