Phosphorylated Derivatives of Transportan 10 for Cellular Delivery of Oligonucleotides (2011)
Sequence: Stearyl-AGYLLGKINLKALAALAKKIL-NH2
splice-correcting oligonucleotide (SCO)
| Experiment Id | EXP001602 |
|---|---|
| Paper | Phosphorylated Derivatives of Transportan 10 for Cellular Delivery of Oligonucleotides |
| Peptide | PepFect3 (PF3) |
| 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 | 1.4 |
| Rna Concentration | 0.2 |
| Mixing Ratio | 7:1 (peptide:ON molar ratio); also tested 1:1–15:1 |
| Formulation Format | non-covalent peptide:ON nanocomplexes (co-incubation) |
| Formulation Components | stearylated peptide + SCO (phosphorothioate 2'-O-methyl ON) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | HeLa pLuc 705 |
| Animal Model | |
| Administration Route | |
| Output Type | splice correction (luciferase activity) |
| Output Value | Functional splice correction observed |
| Output Units | |
| Output Notes | Moderate splice correction; chloroquine significantly increases effect, suggesting endosomal entrapment without assistance |
| Toxicity Notes | Not highlighted as most efficient; used as comparator |
| Curation Notes |