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EXP000977

Paper

Intracellular delivery of therapeutic antisense oligonucleotides targeting mRNA coding mitochondrial proteins by cell-penetrating peptides (2020)

Peptide

mtCPP1 + PF14

Sequence: mtCPP1 (rYaOF-NH2) + PF14 (Stearyl-AGYLLGKLLOOLAAAALOOLL-NH2)

RNA

ASO

All experiment fields

Experiment Id EXP000977
Paper Intracellular delivery of therapeutic antisense oligonucleotides targeting mRNA coding mitochondrial
Peptide mtCPP1 + PF14
Delivery Success Class no
In Vivo Flag no
Uptake Confirmed yes
Label Confidence high
In Vitro Functional Effect yes
Endosomal Escape Evidence
Peptide Concentration 0.3–1.0 µM (depends on MR; oligo 100 nM)
Rna Concentration 100 nM
Mixing Ratio MR10:1 used for bio readouts (also MR3/MR7 for DLS)
Formulation Format non-covalent peptide/oligonucleotide nanocomplex
Formulation Components Peptide + oligonucleotide in MilliQ water (10% final volume), incubated 1 h RT; added to cells in complete medium
Size Nm 20.30
Zeta Mv 21.40
Model Scope in_vitro
Model Type in vitro
Cell Lines Or Primary Cells HeLa pLuc705
Animal Model
Administration Route
Output Type mitochondrial membrane potential (TMRE) + RT-qPCR
Output Value TMRE uptake decreased at 6 h (depolarization) and ROS increased; RT-qPCR: COXII RNA strongly reduced (mitFect3 ~near knockout)
Output Units
Output Notes Mitochondrial antisense activity supported by TMRE/ROS and RT-qPCR (COXII decrease; UCP2 unchanged).
Toxicity Notes WST-1: >80% cell viability (tested MRs)
Curation Notes