Sequence: RXRBRBRXRBRBRBRXX
PMO (morpholino antisense oligo)
| Experiment Id | EXP001206 |
|---|---|
| Paper | Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of |
| Peptide | CPP-I (PPMO-I) |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | 12 mg/kg/day (PPMO dose) for 4 days |
| Mixing Ratio | |
| Formulation Format | covalent peptide–PMO conjugate (PPMO) |
| Formulation Components | CPP-I conjugated to PMO-654 |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | EGFP-654 transgenic mouse |
| Administration Route | intraperitoneal (IP) |
| Output Type | splice correction / EGFP upregulation |
| Output Value | |
| Output Units | |
| Output Notes | RT-PCR splice correction across multiple tissues 1 day after last injection; tissue-specific % correction reported in Fig. 1b. |
| Toxicity Notes | Serum AST/ALT monitored; no overt toxicity reported at tested doses (see Fig. 1c). |
| Curation Notes |