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EXP001694

Paper

In Vitro Investigations of the Efficacy of Cyclodextrin-siRNA Complexes Modified with Lipid-PEG-Octaarginine: Towards a Formulation Strategy for Non-viral Neuronal siRNA Delivery (2013)

Peptide

Octaarginine (R8) in DSPE-PEG2000-R8 (post-inserted)

Sequence: Ac-Cys-βAla-(Arg)8-H (R8 peptide used to synthesize DSPE-PEG2000-R8)

RNA

siRNA

All experiment fields

Experiment Id EXP001694
Paper In Vitro Investigations of the Efficacy of Cyclodextrin-siRNA Complexes Modified with Lipid-PEG-Octa
Peptide Octaarginine (R8) in DSPE-PEG2000-R8 (post-inserted)
Delivery Success Class no
In Vivo Flag yes
Uptake Confirmed yes
Label Confidence high
In Vitro Functional Effect yes
Endosomal Escape Evidence
Peptide Concentration 20 mol% DSPE-PEG2000-R8 relative to CD in complexes (post-inserted; incubated ~18 h at 38°C)
Rna Concentration 50 nM (uptake + luciferase knockdown); 100 nM (GAPDH knockdown + toxicity)
Mixing Ratio CD:siRNA mass ratio (MR) = 20; DSPE-PEG2000-R8 added at 20 mol% (relative to CD)
Formulation Format Cyclodextrin/siRNA nanoparticles with post-inserted DSPE-PEG2000-R8 (R8-PEG-lipid surface layer)
Formulation Components SC12CDclickpropylamine (amphiphilic cationic β-CD) + siRNA; DSPE-PEG2000-R8 micelles post-inserted
Size Nm 113.70
Zeta Mv 38.90
Model Scope in_vivo
Model Type in vitro
Cell Lines Or Primary Cells mHypoE N41 (mouse embryonic hypothalamic neuronal cell line)
Animal Model
Administration Route
Output Type uptake + gene knockdown
Output Value Uptake: 69.3±0.2% FAM+ cells (50 nM, 24 h). Luciferase: ~80% knockdown (50 nM). GAPDH: ~40% reduction (100 nM).
Output Units
Output Notes Complexes stable to salt (OptiMEM) vs unmodified CD complexes; serum (FBS) induced aggregation remained. Controls included NH2-PEG-DSPE (no R8) which did not significantly knock down luciferase.
Toxicity Notes MTT: ~74% dehydrogenase activity for MR20 20% R8 (100 nM siRNA, 24 h); higher R8-PEG-DSPE caused more toxicity.
Curation Notes