Sequence: proprietary / undisclosed
PMO (antisense oligonucleotide)
| Experiment Id | EXP002368 |
|---|---|
| Paper | A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers |
| Peptide | RC-1001 CPP (proprietary) |
| 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 | 10–80 mg/kg (reported doses) |
| Mixing Ratio | |
| Formulation Format | PPMO (peptide–PMO conjugate) |
| Formulation Components | CPP–PMO conjugate (RC-1001) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | mdx mouse (Duchenne muscular dystrophy model) |
| Administration Route | intravenous (tail vein) |
| Output Type | exon skipping, dystrophin restoration, functional rescue |
| Output Value | Up to ~99% exon skipping; up to ~39% dystrophin (quadriceps); improved grip strength and rotarod |
| Output Units | |
| Output Notes | Dose-dependent exon skipping, protein restoration, durability up to 60 days, functional recovery |
| Toxicity Notes | No significant toxicity; well tolerated |
| Curation Notes |