Pip6a-PMO-Ctrl (reverse GAC7 control)
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 | EXP001687 |
|---|---|
| Paper | Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-deri |
| Peptide | Pip6a-PMO-Ctrl (reverse GAC7 control) |
| Delivery Success Class | no |
| In Vivo Flag | yes |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | Dose matched to Pip6a-PMO regimen (reported as control conjugate) |
| 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 |
| Administration Route | Intravenous (tail vein) |
| Output Type | in vivo negative control |
| Output Value | No correction of splicing defects or myotonia in HSA-LR mice. |
| Output Units | |
| Output Notes | Systemic injections of Pip6a-Ctrl had no effect on DM1-splicing defects or myotonia (control conjugate). |
| Toxicity Notes | |
| Curation Notes |