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 | EXP001685 |
|---|---|
| Paper | Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-deri |
| Peptide | Pip6a-PMO-CAG7 |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | |
| 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_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | Patient-derived DM1 differentiated myoblasts (immortalized; 2600 CTG; also 1300 CTG line in supplemental) |
| Animal Model | |
| Administration Route | |
| Output Type | in vitro splice correction / RNA foci reduction |
| Output Value | Significant correction of MBNL1-dependent splicing defects (e.g., LDB3, MBNL1, SOS1, DMD) and reduction of nuclear RNA foci; decreased mutant DMPK transcript levels in DM1 myoblasts after 24 h. |
| Output Units | |
| Output Notes | Differentiated immortalized DM1 myoblasts (2600 CTG repeats) treated with 1 µM Pip6a-PMO for 24 h; FISH/immunofluorescence shows MBNL1 relocation and fewer CUGexp RNA foci; RT-PCR shows splice correction; Northern blot shows reduced mutant DMPK. |
| Toxicity Notes | |
| Curation Notes |