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EXP001419

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 EXP001419
Paper Mitochondrial transgene expression via an artificial mitochondrial DNA vector in cells from a patien
Peptide KALA
Delivery Success Class no
In Vivo Flag yes
Uptake Confirmed no
Label Confidence high
In Vitro Functional Effect
Endosomal Escape Evidence
Peptide Concentration Surface display: STR-KALA 10 mol% of total lipids
Rna Concentration 1 µg pDNA encapsulated in carrier in 50 µL HEPES buffer (pH 7.4)
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_vivo
Model Type in vivo
Cell Lines Or Primary Cells
Animal Model Mice (Balb/c used for biocompatibility evaluation in this study; serum markers at 24 h)
Administration Route Intravenous (tail vein)
Output Type Serum biochemical markers (AST, ALT, BUN, CRE, etc.) at 24 h post-injection
Output Value
Output Units
Output Notes No substantial differences vs saline controls reported for serum biochemical markers after KALA-MITO-Porter administration.
Toxicity Notes Biocompatibility assessment; no major marker elevations reported.
Curation Notes