Sequence: TPA-mPEG-rllrkrlkrlhsalrvrl
| Experiment Id | EXP000534 |
|---|---|
| Paper | Efficient systemic CNS delivery of a therapeutic antisense oligonucleotide with a blood-brain barrie |
| Peptide | BPP8 |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | 8 mg/kg (BPP8); 4 mg/kg (BPP9); 10 mg/kg (BPP12) |
| Mixing Ratio | |
| Formulation Format | Systemic peptide–PMO conjugate |
| Formulation Components | ApoE- or THR-derived BPP–PMO conjugate |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | SMN2+/- transgenic mice (adult) |
| Administration Route | IV (tail vein) |
| Output Type | In vivo splice correction; biodistribution imaging |
| Output Value | BPP8 significantly increased SMN2 in brain and spinal cord; others ineffective |
| Output Units | |
| Output Notes | Only BPP8 demonstrated effective BBB penetration and CNS antisense activity |
| Toxicity Notes | No hepatotoxicity or immune activation for BPP8 |
| Curation Notes |