Sequence: QLFPLFR (cyclized 7-mer; CyPep10 / CP10)
PMO (phosphorodiamidate morpholino oligomer)
| Experiment Id | EXP001569 |
|---|---|
| Paper | Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mo |
| Peptide | CyPep10 (CP10) |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | 30 mg/kg PMO (or molar equivalent), IV weekly for 4 weeks; analysis 1 week after last dose |
| Rna Concentration | 30 mg/kg PMO (or molar equivalent), IV weekly for 4 weeks; analysis 1 week after last dose |
| Mixing Ratio | Covalent conjugate (1:1 peptide:PMO) |
| Formulation Format | peptide–PMO conjugate (PPMO) |
| Formulation Components | CP10-PMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | mdx mouse (C57BL/10ScSn-Dmdmdx/J), male, treated from 4 weeks old |
| Administration Route | IV (tail vein), weekly x4 |
| Output Type | In vivo exon skipping + dystrophin restoration |
| Output Value | Exon skipping: gastrocnemius ~26%, triceps ~29%, diaphragm ~7%, heart ~0.1%. Dystrophin restoration: triceps ~6%, gastrocnemius ~4%, diaphragm ~0.9%, heart ~0.1%. |
| Output Units | |
| Output Notes | CP10 conjugation increased muscle PMO tissue concentrations vs naked PMO but did not improve exon skipping/dystrophin vs naked PMO (discussed as likely limited nuclear delivery). |
| Toxicity Notes | No significant changes in serum safety markers vs naked PMO (liver/kidney and muscle damage markers). |
| Curation Notes |