Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide (2007)
Sequence: RRRRRRRQIKIWFQNRRMKWKKGGC
| Experiment Id | EXP001676 |
|---|---|
| Paper | Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide |
| Peptide | R6Pen–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 + RT-PCR) |
| Output Value | Slightly higher activity than stable linker; EC50 ~0.7±0.3 µM (RNA level); ~60–70% splice correction at 1 µM by RT-PCR. |
| Output Units | |
| Output Notes | Disulfide vs stable linker comparison shows only modest improvement; indicates linker nature not principal factor. |
| Toxicity Notes | No significant toxicity at 1 µM by PI uptake. |
| Curation Notes |