Sequence: Ac-(RXRRBRRXRYQFLIRXRBRXRB)-COOH
PMO (splice-switching antisense oligonucleotide)
| Experiment Id | EXP002296 |
|---|---|
| Paper | Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atr |
| Peptide | Pip6a |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | yes |
| Label Confidence | very high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | 2.5–10 µg/g (PND0/PND2) |
| Rna Concentration | 2.5–10 µg/g |
| Mixing Ratio | 1:1 covalent conjugate |
| Formulation Format | peptide–PMO conjugate |
| Formulation Components | Pip6a–PMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | Severe SMA mouse model (Taiwanese; Smn1−/−; SMN2) |
| Administration Route | systemic intravenous (facial vein / tail vein) |
| Output Type | survival and phenotypic rescue |
| Output Value | median survival up to 456 days; motor and NMJ rescue |
| Output Units | |
| Output Notes | Bodywide SMN restoration including CNS; dose-dependent survival benefit |
| Toxicity Notes | no liver/kidney toxicity; well tolerated |
| Curation Notes |