Sequence: Ac-R6Gly (acetyl-RRRRRRG) (CPP used; also referred to as RC-100117)
PMO (phosphorodiamidate morpholino oligomer)
| Experiment Id | EXP001566 |
|---|---|
| Paper | Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mo |
| Peptide | CPP (acetyl-R6Gly) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.2–30 µM (dose range tested, 24 h) |
| Rna Concentration | 0.2–30 µM (molar equivalent; 24 h) |
| Mixing Ratio | Covalent conjugate (1:1 peptide:PMO) |
| Formulation Format | peptide–PMO conjugate (PPMO) |
| Formulation Components | CPP-PMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | C2C12 myotubes (mouse) |
| Animal Model | mdx mouse (C57BL/10ScSn-Dmdmdx/J), male, treated from 4 weeks old |
| Administration Route | IV (tail vein), weekly x4 |
| Output Type | Splice correction (exon 23 skipping by RT-PCR) |
| Output Value | Highest exon skipping among tested; ~95.5% exon skipping at 30 µM |
| Output Units | |
| Output Notes | CPP-PMO outperformed CP10-PMO and naked PMO across tested doses in C2C12 cells (24 h). |
| Toxicity Notes | |
| Curation Notes |