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EXP001964

Paper

Effective In Vivo Topical Delivery of siRNA and Gene Silencing in Intact Corneal Epithelium Using a Modified Cell-Penetrating Peptide (2019)

Peptide

Chol-POD + chloroquine / siRNA polyplex

Sequence: CGGGARKKAAKAARKKAAKAARKKAAKAARKKAAKA

RNA

siRNA (siLuc vs NSC4; siGLO)

All experiment fields

Experiment Id EXP001964
Paper Effective In Vivo Topical Delivery of siRNA and Gene Silencing in Intact Corneal Epithelium Using a
Peptide Chol-POD + chloroquine / siRNA polyplex
Delivery Success Class no
In Vivo Flag no
Uptake Confirmed yes
Label Confidence high
In Vitro Functional Effect yes
Endosomal Escape Evidence yes
Peptide Concentration
Rna Concentration
Mixing Ratio 35:1 (POD:siRNA) with 1 µM siRNA; chloroquine 30 µM added 1 h before transfection.
Formulation Format CPP–siRNA polyplex + small-molecule endosomal disruptor
Formulation Components Chol-POD polyplexed with siRNA (35:1) in PBS; free chloroquine added (30 µM).
Size Nm 150.80
Zeta Mv 15.60
Model Scope in_vitro
Model Type in vitro
Cell Lines Or Primary Cells HCE-S human corneal epithelial cells
Animal Model
Administration Route
Output Type In vitro functional RNA effect (luciferase knockdown) + endosomal escape proxy (diffuse cytoplasmic siGLO pattern)
Output Value Significant luciferase knockdown observed only when chloroquine was used with the polyplexes; siGLO fluorescence becomes more diffuse vs punctate.
Output Units
Output Notes Used to demonstrate endosomal entrapment barrier and that escape enables RNAi activity.
Toxicity Notes Chloroquine noted as toxic for in vivo use; therefore replaced by covalently attached analog for in vivo.
Curation Notes