Sequence: Ac-R6Gly (acetyl-RRRRRRG) (CPP used; also referred to as RC-100117)
PMO (phosphorodiamidate morpholino oligomer)
| Experiment Id | EXP001570 |
|---|---|
| Paper | Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mo |
| Peptide | CPP (acetyl-R6Gly) |
| 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 | 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 ~51%, triceps ~49%, diaphragm ~25%, heart ~12%. Dystrophin restoration: gastrocnemius ~17%, triceps ~17%, diaphragm ~2%, heart ~0.1%. |
| Output Units | |
| Output Notes | CPP-PMO produced significantly higher exon skipping and dystrophin restoration than naked PMO and CP10-PMO in vivo. |
| Toxicity Notes | No significant differences in general liver/kidney function vs naked PMO; muscle damage biomarkers (AST/ALT/CK/LDH) decreased vs naked PMO in some measures. |
| Curation Notes |