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EXP001417

Paper

Mitochondrial transgene expression via an artificial mitochondrial DNA vector in cells from a patient with a mitochondrial disease (2018)

Peptide

KALA

Sequence: WEKLAKALAKALAKHLAKALAKALKA-NH2

RNA

pDNA (plasmid DNA)

All experiment fields

Experiment Id EXP001417
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); 3 h serum-free then 21 h with serum
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
Formulation Format KALA-MITO-Porter (liposome-based MITO-Porter modified with STR-KALA)
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%)
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 KALA-MITO-Porter produced very low luciferase activity in G625A fibroblasts (~1500 RLU/mg protein) and cellular uptake was significantly lower than in HeLa.
Toxicity Notes
Curation Notes