Sequence: Ac-(RXRRBRRXRYQFLIRXRBRXRB)-COOH
PMO (splice-switching antisense oligonucleotide)
| Experiment Id | EXP002295 |
|---|---|
| Paper | Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atr |
| Peptide | Pip6a |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 25–500 nM |
| Rna Concentration | 25–500 nM |
| Mixing Ratio | 1:1 covalent conjugate |
| Formulation Format | peptide–PMO conjugate |
| Formulation Components | Pip6a–PMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | SH-SY5Y human neuroblastoma cells |
| Animal Model | |
| Administration Route | cell incubation |
| Output Type | splice correction |
| Output Value | ↑ full-length SMN2 transcript |
| Output Units | |
| Output Notes | Dose-dependent increase in FLSMN2; naked PMO inactive |
| Toxicity Notes | no toxicity reported |
| Curation Notes |