L-CP10-CPP (linear double conjugate)
Sequence: CP10 + CPP (linear double conjugation onto PMO)
| Experiment Id | EXP001466 |
|---|---|
| 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 | 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 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_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 + pathology |
| Output Value | Exon skipping: gastrocnemius ~55%, diaphragm ~50%, heart ~20%. Dystrophin restoration: gastrocnemius ~26%, diaphragm ~10%, heart ~3%. Pathology in gastrocnemius reduced (~1.7% vs ~10.8% for naked PMO). |
| Output Units | |
| Output Notes | Linear double conjugate generally outperformed naked PMO and CP10-PMO, and modestly exceeded CPP-PMO in several tissues; also increased tissue PMO concentrations (ELOHA). |
| Toxicity Notes | No significant differences in general liver/kidney function vs naked PMO; muscle damage markers tended lower vs naked PMO. |
| Curation Notes |