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EXP001262

Paper

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

Peptide

EEV2

Sequence: EEV1-derived cyclic CPP + SV40 NLS (PKKKRKV); full sequence not disclosed

RNA

PMO (antisense oligonucleotide)

All experiment fields

Experiment Id EXP001262
Paper The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of
Peptide EEV2
Delivery Success Class no
In Vivo Flag no
Uptake Confirmed no
Label Confidence high
In Vitro Functional Effect yes
Endosomal Escape Evidence
Peptide Concentration
Rna Concentration 1–5 µM
Mixing Ratio
Formulation Format covalent peptide–PMO conjugate
Formulation Components EEV2-PMO-PAS
Size Nm
Zeta Mv
Model Scope in_vitro
Model Type in vitro
Cell Lines Or Primary Cells FSHD donor immortalized myoblasts differentiated to myotubes (AB1080)
Animal Model
Administration Route
Output Type gene expression knockdown
Output Value Reduced ZSCAN4 (and TRIM43) transcript expression toward healthy levels
Output Units
Output Notes EEV2-PMO-PAS added during differentiation; harvested after 5 days; qRT-PCR readout.
Toxicity Notes
Curation Notes