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EXP001334

Paper

Improved cell-penetrating peptide–PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle (2008)

Peptide

RXR4–PNA705

Sequence: (R-Ahx-R)4-C

RNA

PNA (steric-blocking)

All experiment fields

Experiment Id EXP001334
Paper Improved cell-penetrating peptide–PNA conjugates for splicing redirection in HeLa cells and exon ski
Peptide RXR4–PNA705
Delivery Success Class no
In Vivo Flag no
Uptake Confirmed no
Label Confidence medium
In Vitro Functional Effect yes
Endosomal Escape Evidence
Peptide Concentration
Rna Concentration
Mixing Ratio
Formulation Format covalent peptide–PNA conjugate (disulfide or thioether linkage)
Formulation Components HeLa pLuc705 splice-correction assay; conjugates incubated 4 h in OptiMEM then 20 h in complete medium; readouts: luciferase upregulation and RT-PCR splice correction. PNA cargo: Cys-Lys-CCTCTTACCTCAGTTACA-Lys-amide (PNA705, 18-mer). RXR4 is (R-Ahx-R)4-based CPP series used as comparator.
Size Nm
Zeta Mv
Model Scope in_vitro
Model Type in vitro
Cell Lines Or Primary Cells HeLa pLuc705 cells
Animal Model
Administration Route
Output Type EC50 (RT-PCR splice correction, µM)
Output Value 3.5
Output Units
Output Notes Main text indicates RXR4–PNA705 substantially less active; EC50 reported ~3–4 µM in RT-PCR splice correction.
Toxicity Notes
Curation Notes