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EXP000265

Paper

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

Peptide

Myr-HA2-ApoE(133–150)

Sequence: Myr-GLFHAIAHFIHGGWH-X-LRVRLASHLRKLRKRLLR

RNA

ASO (PMO)

All experiment fields

Experiment Id EXP000265
Paper Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and C
Peptide Myr-HA2-ApoE(133–150)
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 Myr-HA2-ApoE(133–150)-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