Sequence: EEV1-derived cyclic CPP + SV40 NLS (PKKKRKV); full sequence not disclosed
PMO (antisense oligonucleotide)
| Experiment Id | EXP001259 |
|---|---|
| Paper | The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of |
| Peptide | EEV2 |
| 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 | 20 mg/kg (PMO equiv) i.v. (single dose) |
| Mixing Ratio | |
| Formulation Format | covalent peptide–PMO conjugate |
| Formulation Components | EEV2-PMO-23 |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | mdx mice (Duchenne muscular dystrophy model) |
| Administration Route | intravenous (i.v.) |
| Output Type | exon skipping / dystrophin restoration |
| Output Value | High exon 23 skipping (e.g., ~61% heart, ~79% diaphragm at 1 week) and dystrophin restoration (~11% heart at 1 week) |
| Output Units | |
| Output Notes | Durable exon skipping up to at least 4 weeks; dystrophin detected by capillary electrophoresis and localized by IHC. |
| Toxicity Notes | |
| Curation Notes |