Peptide Nanoparticle Delivery of Charge-Neutral Splice-Switching Morpholino Oligonucleotides (2015)
Sequence: Stearoyl-AGYLLGKLLOOLAAAALOOLL
PMO (phosphorodiamidate morpholino oligonucleotide; splice-switching)
| Experiment Id | EXP001000 |
|---|---|
| Paper | Peptide Nanoparticle Delivery of Charge-Neutral Splice-Switching Morpholino Oligonucleotides |
| Peptide | PF14 |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | Formulated as nanoparticles; XLA luc reporter assay in U2OS stable cells |
| Rna Concentration | PMO 125–500 nM (U2OS luc reporter) |
| Mixing Ratio | PF14:PMO MR 2:1 and 3:1 (reported); dose series 125–500 nM |
| Formulation Format | noncovalent lipopeptide/PMO nanoparticles (co-incubation; complex formation) |
| Formulation Components | PF14 + PMO XLA (BTK intron reporter splice correction); 5'-CTACAGAGTTCTCAGGATGTAAGCA-3' |
| Size Nm | 134.00 |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | U2OS stable luciferase reporter (BTK intron XLA model); SMA type I fibroblasts GM03813; H2K mdx myotubes + healthy H2K myotubes |
| Animal Model | |
| Administration Route | |
| Output Type | in vitro splice switching (RT-PCR exon inclusion/skipping; luc reporter splice correction) |
| Output Value | Dose-dependent splice correction of luciferase reporter (corrected luc band increased vs naked PMO). |
| Output Units | |
| Output Notes | PF14 nanoparticles active in XLA reporter model. Complexes reported as homogeneous by NTA. |
| Toxicity Notes | Myotube viability (MTS): no toxicity at working concentration (~5 µM peptide). St-RXR4 showed toxicity at 40 µM (without PMO). |
| Curation Notes |