Sequence: LRKLRKRLLR
PMO (splice-switching morpholino)
| Experiment Id | EXP000799 |
|---|---|
| Paper | Amphiphilic lipopeptide significantly enhances uptake of charge-neutral splice switching morpholino |
| Peptide | ApoE (CPP fragment) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | ~0.5 µM (as peptide–PMO conjugate) + GMO 8 µg/mL |
| Rna Concentration | ~0.5 µM (PMO equivalent) |
| Mixing Ratio | |
| Formulation Format | cubosome formulation (GMO) |
| Formulation Components | GMO (monoolein) + Pluronic F108 + ApoE-PMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | SMA Type I patient-derived fibroblasts (GM03813) |
| Animal Model | |
| Administration Route | |
| Output Type | splice-switching activity (RT-PCR; % full-length SMN2 transcript) |
| Output Value | No statistically significant improvement vs ApoE-PMO alone |
| Output Units | |
| Output Notes | GMO cubosomes reduced viability above 10 µg/mL; 8 µg/mL used for loading; SAXS indicates cubic phase disrupted upon PA-PMO addition. |
| Toxicity Notes | GMO caused reduced viability above 10 µg/mL; 8 µg/mL used for functional testing. |
| Curation Notes |