CPP-B conjugate (M23D-B, dystrophin-targeting PPMO)
Sequence: RXRRBRRXRRBRXB
PMO (morpholino antisense oligo)
| Experiment Id | EXP001212 |
|---|---|
| Paper | Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of |
| Peptide | CPP-B conjugate (M23D-B, dystrophin-targeting PPMO) |
| 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-B conjugated to PMO M23D (exon 23 skipping) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | mdx mouse (DMD model) |
| Administration Route | intravenous (IV) |
| Output Type | exon skipping / dystrophin restoration / pathology biomarkers |
| Output Value | Sustained exon 23 skipping; dystrophin restoration in skeletal + cardiac muscle; serum CK markedly reduced. |
| Output Units | |
| Output Notes | Nested RT-PCR showed exon 23 skipping in heart/diaphragm/quadriceps up to 9 weeks; dystrophin detected by in-gel western and immunofluorescence; reduced inflammatory infiltration in heart (Figs. 3–5). |
| Toxicity Notes | No detectable toxicity reported; clinical chemistry (BUN/creatinine/AST/ALT/CK) monitored. |
| Curation Notes |