Back to browse

EXP001332

Paper

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

Peptide

Pip2a–PNA705

Sequence: (R-Ahx-R)3-IdKILFQN-dRRMKWHKBC

RNA

PNA (steric-blocking)

All experiment fields

Experiment Id EXP001332
Paper Improved cell-penetrating peptide–PNA conjugates for splicing redirection in HeLa cells and exon ski
Peptide Pip2a–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). Serum-stabilized Pip peptide (candidate for in vivo).
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 0.79
Output Units
Output Notes Supplementary Table 1 lists Pip2a–PNA705 EC50 = 0.79 ± 0.13 µM; predominantly stable 1 h in 20% mouse serum.
Toxicity Notes
Curation Notes