Sequence: Ac-RXRRBRRXRRBRXB (B peptide; X = 6-aminohexanoic acid, B = beta-alanine)
| Experiment Id | EXP002442 |
|---|---|
| Paper | Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in Btk |
| Peptide | B-PMO 186 |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | medium |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.625–5 µM in reporter-cell assay; 0.3 and 5 µM in primary B-cell ex vivo assay |
| Rna Concentration | |
| Mixing Ratio | |
| Formulation Format | covalent peptide-oligonucleotide conjugate |
| Formulation Components | B peptide + PMO SSO 186 |
| Size Nm | |
| Zeta Mv | |
| Model Scope | ex_vivo |
| Model Type | in vitro / ex vivo |
| Cell Lines Or Primary Cells | U2OS luciferase BTK intron 4 reporter cells; primary B cells from BAC transgenic mouse |
| Animal Model | |
| Administration Route | cell incubation |
| Output Type | splice correction / BTK restoration |
| Output Value | Reporter-cell splice correction was lower than Pip6a-PMO 186; primary B cells showed minor BTK restoration relative to Pip6a 186 conjugates |
| Output Units | |
| Output Notes | Included as a peptide-conjugated comparator system; no in vivo phenotypic rescue testing in this paper |
| Toxicity Notes | Comparator peptide-PMO system; exact B-peptide and PMO 186 sequences updated from supplementary information |
| Curation Notes |