Sequence: AcRXRRBRRFQILYRBRXRB
| Experiment Id | EXP001028 |
|---|---|
| Paper | Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBN |
| Peptide | Pip9b2 |
| 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 | ~9.2 mg/kg (IV, biweekly ×3; 150 µL of 1.875 mg/mL solution) |
| Mixing Ratio | |
| Formulation Format | covalent conjugate (CPP-PMO) |
| Formulation Components | Pip9b2 (CPP) maleimide-linked to PMO antimiR |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | HSALR transgenic mouse model of DM1 (line 20b); males ~5–5.5 months |
| Administration Route | Intravenous (biweekly; 3 injections; sacrifice day 45) |
| Output Type | miRNA inhibition + Mbnl1 upregulation + splicing correction / myotonia improvement |
| Output Value | |
| Output Units | |
| Output Notes | CPP-PMO antimiRs showed in vivo target engagement in skeletal muscle and improved DM1 molecular/functional phenotypes; scrambled control showed no miRNA inhibition. |
| Toxicity Notes | No significant weight or spleen-weight changes reported; cytokine/immune activation markers not increased vs controls. |
| Curation Notes |