Sequence: Ac-RXRRBRRXRYQFLIRXRBRXRB (Pip6a; X = 6-aminohexanoic acid, B = beta-alanine)
| Experiment Id | EXP002443 |
|---|---|
| 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 + PGO 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 | Pip6a conjugation markedly increased PGO activity; Pip6a-PGO-186 gave efficient BTK protein restoration in primary B cells |
| Output Units | |
| Output Notes | Neutral PGO chemistry alone had low activity, but Pip6a conjugation increased activity to a level similar to Pip6a-PMO in vitro |
| Toxicity Notes | Pip6a-PGO-186 sequence updated from supplementary information; lowercase bases are 2′-O-methyl RNA and * indicates PGO/Dmi linkages |
| Curation Notes |