Sequence: EEV1-derived cyclic CPP + SV40 NLS (PKKKRKV); full sequence not disclosed
PMO (antisense oligonucleotide)
| Experiment Id | EXP001261 |
|---|---|
| Paper | The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of |
| Peptide | EEV2 |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | 1–10 µM |
| Mixing Ratio | |
| Formulation Format | covalent peptide–PMO conjugate |
| Formulation Components | EEV2-PMO-PAS |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | FSHD patient-derived lymphoblastoid cells (GM16283) |
| Animal Model | |
| Administration Route | |
| Output Type | gene expression knockdown |
| Output Value | Dose-dependent reduction of DUX4 downstream targets (e.g., MBD3L2, ZSCAN4, TRIM43) by qRT-PCR |
| Output Units | |
| Output Notes | Compared vs untreated FSHD and healthy donor lymphoblastoid cells. |
| Toxicity Notes | |
| Curation Notes |