Sequence: Ac-RXRRBRRXRYQFLIRXRBRXRB (Pip6a; X = 6-aminohexanoic acid, B = beta-alanine)
| Experiment Id | EXP002441 |
|---|---|
| Paper | Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in Btk |
| Peptide | Pip6a-PMO 186 |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.625–5 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 | Pip6a cell-penetrating 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 and BTK protein restoration |
| Output Value | Reporter cells: strong luciferase rescue and corrected BTK RNA; primary B cells: detectable BTK protein after 48 h |
| Output Units | |
| Output Notes | Pip6a-PMO 186 had superior activity over B-PMO 186 and was one of the most efficient ex vivo conjugates |
| Toxicity Notes | Reporter and primary B-cell data; exact Pip6a and PMO 186 sequences updated from supplementary information |
| Curation Notes |