Sequence: GLFHAIAHFIHGGWH-LRVRLASHLRKLRKRLLR
| Experiment Id | EXP000272 |
|---|---|
| Paper | Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and C |
| Peptide | HA2-ApoE(133–150) |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | yes |
| Label Confidence | very high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 8 mg/kg |
| Rna Concentration | Equimolar |
| Mixing Ratio | 1:1 covalent conjugate |
| Formulation Format | Peptide–PMO conjugate |
| Formulation Components | HA2-ApoE(133–150)-PMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | SMN2 transgenic mice |
| Administration Route | Intravenous (tail vein) |
| Output Type | SMN2 exon-7 inclusion (RT-qPCR) |
| Output Value | Significant increase in brain and spinal cord |
| Output Units | |
| Output Notes | Demonstrated BBB crossing and CNS delivery |
| Toxicity Notes | Well tolerated at 8 mg/kg |
| Curation Notes |