Sequence: N-(RXRRBRRXRRBRXB)-C (B=β-alanine; X=6-aminohexanoic acid)
PMO (antisense oligonucleotide)
| Experiment Id | EXP001280 |
|---|---|
| Paper | Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac d |
| Peptide | B peptide |
| 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 | 6 mg/kg weekly ×3 (i.v.; total 18 mg/kg) |
| 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 | Dystrophin restoration (western blot) |
| Output Value | ~10% dystrophin in TA/quadriceps/gastrocnemius; <10% abdominal/diaphragm; undetectable in heart |
| Output Units | |
| Output Notes | At 6 mg/kg weekly ×3, B-PMO shows low dystrophin restoration and none detectable in heart (Supplementary Fig. 2C). |
| Toxicity Notes | No toxicity reported at this dose regime. |
| Curation Notes |