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EXP001252

Paper

The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders (2023)

Peptide

EEV1

Sequence: Proprietary cyclic CPP (sequence not disclosed)

RNA

PMO (antisense oligonucleotide)

All experiment fields

Experiment Id EXP001252
Paper The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of
Peptide EEV1
Delivery Success Class no
In Vivo Flag no
Uptake Confirmed yes
Label Confidence high
In Vitro Functional Effect yes
Endosomal Escape Evidence
Peptide Concentration
Rna Concentration 0.6 µM (PMO equiv; also tested up to 10 µM)
Mixing Ratio
Formulation Format covalent peptide–PMO conjugate
Formulation Components EEV1-PMO654
Size Nm
Zeta Mv
Model Scope in_vitro
Model Type in vitro
Cell Lines Or Primary Cells HeLa EGFP-654 cells
Animal Model
Administration Route
Output Type splice correction / reporter expression
Output Value Dose-dependent EGFP restoration; 0.6 µM EEV1-PMO654 ~ similar to 20 µM naked PMO654
Output Units
Output Notes EGFP restored by flow cytometry and live-cell fluorescence microscopy; splice switching shown by RT-PCR.
Toxicity Notes
Curation Notes