Sequence: AcKXKKBKKFQILYKBKXKC-(NH2)Ma.HEX
| Experiment Id | EXP001029 |
|---|---|
| Paper | Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBN |
| Peptide | Pip9b2KC |
| Delivery Success Class | no |
| In Vivo Flag | yes |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | 0.4 nM–5 µM (dose range); gymnosis at EC50 |
| Mixing Ratio | |
| Formulation Format | covalent conjugate (CPP-PMO) |
| Formulation Components | Pip9b2KC (CPP) maleimide-linked to PMO antimiR |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | Immortalized MyoD-inducible DM1 fibroblasts transdifferentiated to myoblasts/myotubes |
| Animal Model | |
| Administration Route | |
| Output Type | MBNL1 protein upregulation (QDB) |
| Output Value | |
| Output Units | |
| Output Notes | Assessed by lipofection and gymnosis; CPP-PMO antimiRs increased MBNL1 levels vs untreated DM1 cells. Some compounds showed activity by gymnosis (no transfection reagent). |
| Toxicity Notes | Gymnosis generally showed a more favorable cell viability profile than transfection reagent. |
| Curation Notes |