Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide (2007)
Sequence: RRRRRRRRRRQIKIWFQNRRMKWKKGGC
| Experiment Id | EXP001684 |
|---|---|
| Paper | Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide |
| Peptide | R9Pen–PNA705 (disulfide) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.1–2.5 µM conjugate tested (typically 1 µM; 4 h incubation in OptiMEM) |
| Rna Concentration | Same as conjugate (PNA is covalently linked); 0.1–2.5 µM |
| Mixing Ratio | Covalent conjugate (1:1 peptide:PNA) |
| Formulation Format | CPP–PNA conjugate (thioacetyl stable linker or disulfide linker) |
| Formulation Components | CPP peptide covalently conjugated to PNA705 (or scrambled) (± chloroquine 100 µM in some conditions) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | HeLa pLuc705 splice-correction reporter cells |
| Animal Model | |
| Administration Route | |
| Output Type | splice correction (luciferase) |
| Output Value | Enhanced vs Pen at 1 µM; R6 tail was optimal among z=0/3/6/9 (Figure 5). |
| Output Units | |
| Output Notes | Arg tail length modulates activity; z=6 best at 1 µM. |
| Toxicity Notes | No significant toxicity at 1 µM by PI uptake (reported for series). |
| Curation Notes |