L-CP10-CPP (linear double conjugate)
Sequence: CP10 + CPP (linear double conjugation onto PMO)
PMO (phosphorodiamidate morpholino oligomer)
| Experiment Id | EXP001567 |
|---|---|
| Paper | Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mo |
| Peptide | L-CP10-CPP (linear double conjugate) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 5–30 µM (tested; compared to CPP-PMO) |
| Rna Concentration | 5–30 µM (molar equivalent) |
| Mixing Ratio | Covalent double conjugate (CP10 + CPP on one PMO; linear) |
| Formulation Format | double peptide–PMO conjugate (PPMO) |
| Formulation Components | L-CP10-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 | At 5 µM achieved ~40% exon skipping vs ~20% for CPP-PMO; at 15 µM reached ~90% (similar to CPP-PMO at 30 µM) |
| Output Units | |
| Output Notes | Double-conjugated PMOs enabled high exon skipping at lower doses compared with CPP-PMO alone in vitro. |
| Toxicity Notes | |
| Curation Notes |