Sequence: 2-branched dendrimer; total 16 Arg; each arm ~RXRRXRRXRRXR; C-terminal Cys
| Experiment Id | EXP001600 |
|---|---|
| Paper | Synthesis and Splice-Redirecting Activity of Branched, Arginine-Rich Peptide Dendrimer Conjugates of |
| Peptide | RXR8-2B |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 1 µM |
| Rna Concentration | 1 µM (1:1 conjugate) |
| Mixing Ratio | |
| Formulation Format | covalent peptide–PMO conjugate |
| Formulation Components | RXR8-2B–PMO (exon 23) conjugate |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | Differentiated mdx mouse muscle cells (exon 23 skipping assay) |
| Animal Model | |
| Administration Route | |
| Output Type | exon skipping |
| Output Value | >70% exon 23 skipping (in 10% FBS; no transfection agent) |
| Output Units | |
| Output Notes | Differentiated mdx mouse muscle cells; incubated with 1 µM conjugate in 10% FBS; exon skipping reported. |
| Toxicity Notes | Not reported for mdx muscle cells (in vitro). |
| Curation Notes |