Sequence: Proprietary cyclic CPP (sequence not disclosed)
PMO (antisense oligonucleotide)
| Experiment Id | EXP001255 |
|---|---|
| Paper | The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of |
| Peptide | EEV1 |
| 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 | 10 mg/kg (PMO equiv) ×4 daily |
| Mixing Ratio | |
| Formulation Format | covalent peptide–PMO conjugate |
| Formulation Components | EEV1-PMO654 |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | EGFP-654 transgenic mice |
| Administration Route | intravenous (i.v.) |
| Output Type | splice correction |
| Output Value | Splice correction detectable in diaphragm and heart; heart ~10% for EEV1-PMO654 while PMO654 below detection |
| Output Units | |
| Output Notes | Assessed 1 day after last injection by RT-PCR in diaphragm and heart. |
| Toxicity Notes | |
| Curation Notes |