Sequence: N-(RXRRXRRXRRXRXB)-C (X=6-aminohexanoic acid; B=β-alanine)
PMO (antisense oligonucleotide)
| Experiment Id | EXP001277 |
|---|---|
| Paper | Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac d |
| Peptide | P007 |
| 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 | 25 mg/kg PMO-equivalent (single i.v. tail vein injection) |
| Mixing Ratio | covalent conjugate (amide linker) |
| Formulation Format | peptide–PMO conjugate (covalent) |
| Formulation Components | Arginine-rich CPP peptide + PMO (M23D) via stable amide linker |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | mdx mouse (Duchenne muscular dystrophy model), adult (6–8 weeks) |
| Administration Route | intravenous (tail vein) |
| Output Type | Exon 23 skipping (RT-PCR); dystrophin restoration (immunostaining/western); muscle function (grip strength) |
| Output Value | Skeletal muscle dystrophin ~25–100% of normal; heart ~10–20% of normal at 3 weeks; grip strength improved to normal range |
| Output Units | |
| Output Notes | Single low-dose systemic P007-PMO produced widespread dystrophin restoration across muscles; ~50% exon skipping in heart; grip strength improved. |
| Toxicity Notes | No overt ill health; liver/kidney histology normal; serum AST/ALT reduced toward normal; urea/creatinine unchanged at 25 mg/kg (P007-PMO). |
| Curation Notes |