Sequence: Ac-LRKLRKRLLRXC-NH2 (X = unspecified spacer as shown in paper schematic)
splice-switching PMO (morpholino)
| Experiment Id | EXP001488 |
|---|---|
| Paper | Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outco |
| Peptide | ApoE |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.5–1 µM (P-PMO) |
| Rna Concentration | 0.5–1 µM (PMO, covalent to peptide) |
| Mixing Ratio | 1:1 (covalent conjugate) |
| Formulation Format | covalent CPP–PMO conjugate |
| Formulation Components | ApoE peptide–PMO (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: FL-SMN2 and Δ7-SMN2) ± SMN protein (western blot) |
| Output Value | Increased FL-SMN2 and reduced Δ7-SMN2 vs untreated (splice correction in vitro) |
| Output Units | |
| Output Notes | ApoE-PMO alone promoted exon 7 inclusion in SMN2 transcripts (functional splice switching in SMA fibroblasts). |
| Toxicity Notes | No significant toxicity reported for ApoE-PMO at 0.5–1 µM for 4 h or 16 h (MTS assay). |
| Curation Notes |