Sequence: Pip8b2 (sequence not disclosed in this paper)
| Experiment Id | EXP002448 |
|---|---|
| Paper | Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Swit |
| Peptide | Pip8b2 |
| 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 | 0.1–0.3 µM (each PPMO) |
| Mixing Ratio | PPMO vs PMO |
| Formulation Format | PPMO conjugate |
| Formulation Components | Pip8b2–PMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | Immortalized CXMDJ canine myoblasts |
| Animal Model | |
| Administration Route | |
| Output Type | splice correction / dystrophin restoration |
| Output Value | Markedly enhanced multiexon skipping and dystrophin expression vs naked PMO |
| Output Units | |
| Output Notes | 14-fold lower EC50 compared to PMO |
| Toxicity Notes | No toxicity reported in vitro |
| Curation Notes |