Sequence: CH3CO-RAhxRRAhxRRAhxRRAhxRAhxB-COOH
| Experiment Id | EXP002297 |
|---|---|
| Paper | Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction |
| Peptide | (R-Ahx-R)4 |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.1–10 µM (splice correction dose-response); 2 µM for uptake imaging/flow; up to 40 µM for PI uptake (membrane permeabilization) |
| Rna Concentration | Equimolar to peptide (covalent CPP–PMO705 conjugate); 0.1–10 µM tested |
| Mixing Ratio | |
| Formulation Format | covalent peptide–PMO conjugate |
| Formulation Components | (R-Ahx-R)4–PMO705 conjugate (steric-blocking antisense morpholino) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | HeLa pLuc705 (splice correction luciferase reporter); CHO-K1 and CHO-pgs745 used for proteoglycan-dependent uptake mechanistic assays |
| Animal Model | |
| Administration Route | |
| Output Type | splice correction (luciferase) + uptake |
| Output Value | Robust, dose-dependent splice correction; significant luciferase induction from ~0.1 µM up to 10 µM; RT-PCR shows increased correctly spliced mRNA |
| Output Units | |
| Output Notes | Active without chloroquine (endosomolytic agent); chloroquine further enhances activity; microscopy shows puncta with some diffuse signal over time consistent with escape/nuclear access |
| Toxicity Notes | Very low cytotoxicity; no PI uptake (no detectable membrane permeabilization) up to 40 µM; activity retained (reduced) in 10% serum |
| Curation Notes |