Sequence: Not reported in main text (Pip6a described as hydrophobic core flanked by arginine-rich domains with β-alanine and aminohexanoyl spacers).
PMO (morpholino antisense oligonucleotide)
| Experiment Id | EXP001686 |
|---|---|
| Paper | Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-deri |
| Peptide | Pip6a-PMO-CAG7 |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | Dose: 12.5 mg/kg Pip6a-PMO per injection (1–3 injections; analysis at 2 weeks; durability to 6 months) |
| Rna Concentration | Same as dose (conjugate) |
| Mixing Ratio | Covalent conjugate (1:1 peptide:PMO) |
| Formulation Format | Peptide–PMO conjugate (Pip6a-PMO) |
| Formulation Components | Pip6a peptide covalently conjugated to PMO-CAG7 (or Pip6a-Ctrl control sequence) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | HSA-LR myotonic dystrophy mouse model (250 CTG repeats in HSA transgene) |
| Administration Route | Intravenous (tail vein) |
| Output Type | in vivo splice correction + phenotypic rescue |
| Output Value | Near-complete correction of DM1 splicing defects (Clcn1 exon 7a, Mbnl1 exon 5, Atp2a1 exon 22) after 2–3 injections (12.5 mg/kg) and complete abolition of myotonia; effects persisted up to ~6 months. |
| Output Units | |
| Output Notes | HSA-LR mice received IV (tail vein) injections: single 12.5 mg/kg (partial correction) and 2–3 injections 12.5 mg/kg (almost complete/full correction). Functional myotonia assay normalized; transcriptomic and proteomic shifts toward WT reported. |
| Toxicity Notes | Mild liver/kidney histological changes at 2 weeks mostly reversed by 6 months; described as transient/reversible. |
| Curation Notes |