Sequence: WEKLAKALAKALAKHLAKALAKALKA-NH2
| Experiment Id | EXP001418 |
|---|---|
| Paper | Mitochondrial transgene expression via an artificial mitochondrial DNA vector in cells from a patien |
| Peptide | KALA |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | Surface display: STR-KALA 10 mol% of total lipids |
| Rna Concentration | 0.4 µg pDNA per well (24-well plate); RP aptamer 4 mol% used for optimized formulation |
| Mixing Ratio | Protamine condensation N/P=2.3; final DNA concentration for packaging 35 µg/mL; lipid envelope DOPE/SM/CHEMS = 9:2:1 (molar ratio) lipid envelope around protamine-condensed pDNA; STR-KALA 10 mol% of total lipids; Chol-RP 4 mol% of total lipids (optimized; 1–6 mol% screened) |
| Formulation Format | RP/KALA-MITO-Porter (KALA-MITO-Porter additionally modified with Chol-RP aptamer) |
| Formulation Components | Protamine-condensed pDNA core + lipid envelope (DOPE/SM/CHEMS = 9:2:1 (molar ratio) lipid envelope around protamine-condensed pDNA) + STR-KALA (10 mol%) + Chol-RP aptamer (4 mol%) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | G625A patient-derived fibroblasts (mitochondrial disease; tRNA Phe mutation) |
| Animal Model | |
| Administration Route | |
| Output Type | Mitochondrial transgene expression (NanoLuc luciferase activity, RLU/mg protein) |
| Output Value | |
| Output Units | |
| Output Notes | RP (4 mol%) modification increased cellular uptake and enabled strong mitochondrial transgene expression (>20,000 RLU/mg protein) with pCMV-mtLuc(CGG)[hND4]; TAG negative-control plasmid showed no activity. |
| Toxicity Notes | |
| Curation Notes |