Sequence: NH2-RRRRRRRRRFFC-CONH2
| Experiment Id | EXP002299 |
|---|---|
| Paper | Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction |
| Peptide | R9F2 |
| 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 | R9F2–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 | Low splice correction without endosomolytic agent; could not be validly tested at higher concentrations due to toxicity |
| Output Units | |
| Output Notes | Highest uptake by flow cytometry among tested CPP–PMO conjugates but poor splice correction, consistent with endosomal sequestration |
| Toxicity Notes | High cytotoxicity in serum-free medium at 1–10 µM range (limited usable dose range); PI uptake observed |
| Curation Notes |