Sequence: ETWWK
| Experiment Id | EXP001149 |
|---|---|
| Paper | Advancing Antisense Oligonucleotide Delivery through Click Chemistry Based Chemical Conjugation with |
| Peptide | ETWWK |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | yes |
| Peptide Concentration | 1.2 µM (functional); 5 µM used for uptake assays |
| Rna Concentration | 1.2 µM (functional); 5 µM used for uptake assays |
| Mixing Ratio | |
| Formulation Format | covalent CPP–ASO conjugate (CuAAC click chemistry) |
| Formulation Components | ETWWK peptide + exon51 ASO via triazole linker |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | C2C12; human DMD patient-derived myoblasts (KM1327, KM1328); human DMD patient-derived skin fibroblasts |
| Animal Model | |
| Administration Route | |
| Output Type | dystrophin protein expression / splice correction |
| Output Value | Increased dystrophin protein in KM1328 vs unconjugated ASO; Lipofectamine 2000 used as positive control |
| Output Units | |
| Output Notes | Uptake shown by fluorescence microscopy (C2C12) and flow cytometry (KM1328) using FAM-ETWWK-ASO vs FAM-ASO; uptake inhibited by chlorpromazine suggesting clathrin-mediated endocytosis. Lysosomal escape assessed by LysoTracker colocalization (reduced colocalization; nuclear shift). Functional readout: dystrophin upregulation by western blot after 24 h treatment at 1.2 µM. |
| Toxicity Notes | MTT cytotoxicity evaluated in C2C12 and patient-derived myoblast/fibroblast cells; ETWWK-ASO reported as cytocompatible (arginine-containing variants noted as more cytotoxic). |
| Curation Notes |