Sequence: (Ac-pwvpswmpprht-mPEG)2-K-mPEG-hwgghifhaiahflg-K(TPA)
PMO (antisense oligonucleotide)
| Experiment Id | EXP000539 |
|---|---|
| Paper | Efficient systemic CNS delivery of a therapeutic antisense oligonucleotide with a blood-brain barrie |
| Peptide | BPP12 |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | yes |
| Peptide Concentration | |
| Rna Concentration | 0.25–4 µM (in vitro) |
| Mixing Ratio | 3:1 peptide:PMO (molar, conjugation) |
| Formulation Format | Covalent peptide–PMO conjugate |
| Formulation Components | ApoE- or THR-derived BBB-penetrating peptide ± HA2 endosomal escape peptide, covalently linked to PMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | SMA patient-derived fibroblasts (GM03813); HEK293 (toxicity) |
| Animal Model | |
| Administration Route | |
| Output Type | Splice correction (RT-qPCR SMN2 exon-7 inclusion) |
| Output Value | Significant concentration-dependent increase in full-length SMN2 |
| Output Units | |
| Output Notes | All BPP–PMO conjugates active in vitro; ApoE(133–150) strongest |
| Toxicity Notes | Minimal toxicity at effective concentrations |
| Curation Notes |