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EXP001695

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

RNA

siRNA (fluorescently labeled)

All experiment fields

Experiment Id EXP001695
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 no
Label Confidence high
In Vitro Functional Effect
Endosomal Escape Evidence
Peptide Concentration MR20 complexes with 20 mol% DSPE-PEG2000-R8; dose as formulated nanoparticles
Rna Concentration 40 µg FAM-siRNA injected per mouse (formulated or free)
Mixing Ratio CD:siRNA MR=20; 20 mol% DSPE-PEG2000-R8
Formulation Format R8-PEG-DSPE post-inserted CD/siRNA nanoparticles (concentrated by ultrafiltration for PK study)
Formulation Components SC12CDclickpropylamine + FAM-siRNA + DSPE-PEG2000-R8
Size Nm
Zeta Mv
Model Scope in_vivo
Model Type in vivo
Cell Lines Or Primary Cells
Animal Model Male Balb/c mice (8–10 weeks)
Administration Route Intravenous (tail vein)
Output Type pharmacokinetics (plasma)
Output Value Formulated siRNA half-life 15.7±4.4 min vs free siRNA 2.3±0.9 min; ~3× higher plasma levels at 10 min vs free siRNA.
Output Units
Output Notes Plasma FAM-siRNA quantified by fluorescence after extraction; effect described as modest prolongation.
Toxicity Notes
Curation Notes