Sequence: Stearyl-AGYLLGKLLOOLAAAALOOLL-NH2 (O = ornithine)
ASO (antisense oligonucleotide; steric-blocking)
| Experiment Id | EXP001729 |
|---|---|
| Paper | The nuclear concentration required for antisense oligonucleotide activity in myotonic dystrophy cell |
| Peptide | PepFect14 (PF14) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | no |
| Endosomal Escape Evidence | yes |
| Peptide Concentration | Matched to CAG5 condition at same ASO dose (e.g., 9× molar excess). |
| Rna Concentration | Control ASO tested alongside CAG5 (e.g., 0.1–1.0 µM). |
| Mixing Ratio | N/P = 3 (PF14), peptide:ASO molar ratio 9:1; stabilized ~1 h RT. |
| Formulation Format | Noncovalent CPP/ASO polyplex (charge-driven nanoparticle) |
| Formulation Components | PF14 + control steric-blocking ASO (GAC5) polyplex. |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | Immortalized human DM1 myoblasts (DM11 cl5) and unaffected myoblasts (C25); fibroblast validation in some experiments |
| Animal Model | |
| Administration Route | |
| Output Type | in vitro negative control (splice correction) |
| Output Value | No splice correction of DM1-associated mis-splicing compared with untreated DM1 cells. |
| Output Units | |
| Output Notes | Used as sequence control to confirm specificity of CAG5 activity. |
| Toxicity Notes | Same PF14 dose-dependent viability trend applies; control used for interpretation. |
| Curation Notes |