B-CP10-CPP (branched double conjugate)
Sequence: CP10 + CPP (branched double conjugation onto PMO)
| Experiment Id | EXP001467 |
|---|---|
| Paper | Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mo |
| Peptide | B-CP10-CPP (branched 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; branched) |
| Formulation Format | double peptide–PMO conjugate (PPMO) |
| Formulation Components | B-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 |
| Output Value | Exon skipping: gastrocnemius ~37%, diaphragm ~25%, heart ~15%. Dystrophin restoration: gastrocnemius ~17%, diaphragm ~7%, heart ~3%. |
| Output Units | |
| Output Notes | Branched double conjugate improved outcomes vs naked PMO and CP10-PMO; in gastrocnemius/diaphragm it was generally below CPP-PMO, but heart outcomes were comparable or higher in some comparisons; 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 |