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EXP000269

Paper

Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and CNS activity of systemically administered antisense oligonucleotides (2021)

Peptide

Cy5-HA2-ApoE(141–150)

Sequence: Cy5-GLFHAIAHFIHGGWH-X-LRKLRKRLLR

RNA

ASO (PMO)

All experiment fields

Experiment Id EXP000269
Paper Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and C
Peptide Cy5-HA2-ApoE(141–150)
Delivery Success Class no
In Vivo Flag no
Uptake Confirmed yes
Label Confidence medium
In Vitro Functional Effect yes
Endosomal Escape Evidence
Peptide Concentration 0.5–5 µM
Rna Concentration Equimolar
Mixing Ratio 1:1 covalent conjugate
Formulation Format Peptide–PMO conjugate
Formulation Components Cy5-HA2-ApoE(141–150)-PMO
Size Nm
Zeta Mv
Model Scope in_vitro
Model Type in vitro
Cell Lines Or Primary Cells SMA patient-derived fibroblasts (GM03813)
Animal Model
Administration Route
Output Type SMN2 exon-7 inclusion (RT-qPCR)
Output Value Measured
Output Units
Output Notes In vitro screening
Toxicity Notes No toxicity ≤5 µM
Curation Notes