Sequence: N-(RXRRBRRXRRBRXB)-C (B=β-alanine; X=6-aminohexanoic acid)
PMO (antisense oligonucleotide)
| Experiment Id | EXP001276 |
|---|---|
| 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 | 5 µg (local intramuscular TA injection; single dose) |
| 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 | intramuscular (tibialis anterior) |
| Output Type | Dystrophin restoration (immunostaining) |
| Output Value | Near-normal dystrophin-positive fibers locally in TA at 3 weeks |
| Output Units | |
| Output Notes | Local intramuscular delivery shows near normal dystrophin restoration in TA (Supplementary Fig. 1). |
| Toxicity Notes | |
| Curation Notes |