Improving the efficacy of liposome-mediated vascular gene therapy via lipid surface modifications (2017)
Sequence: RRRRRRRR
| Experiment Id | EXP001435 |
|---|---|
| Paper | Improving the efficacy of liposome-mediated vascular gene therapy via lipid surface modifications |
| Peptide | Octaarginine (R8) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | Surface density: 10 mol% STR-R8 of total lipids |
| Rna Concentration | Gene silencing tested at 200–400 nM encapsulated siRNA (GAPDH or NC) in HASMCs; transfection 24 h serum-free then 24 h recovery |
| Mixing Ratio | Transfection efficacy depended on lipid-to-siRNA w:w ratio (2.5:1–20:1) for 10 mol% formulation |
| Formulation Format | R8-modified PEGylated neutral liposomes (R8-PLP) |
| Formulation Components | Neutral PEGylated liposome (PLP) base: DOPC:cholesterol 7:3 mol + 10 mol% DSPE-PEG2000 + STR-R8 (10 mol%) + encapsulated siRNA; Ethanol injection with siRNA (50 µg) in 10 mM Tris-HCl pH 8.0; neutral formulations include 10 mM CaCl2 during assembly; dialyzed and extruded (100 nm) |
| Size Nm | 40.20 |
| Zeta Mv | 10.50 |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | Human aortic smooth muscle cells (HASMCs, primary; 49-year-old male donor) |
| Animal Model | |
| Administration Route | |
| Output Type | In vitro functional effect: GAPDH mRNA knockdown by qPCR (normalized to 18S; compared to control or NC siRNA) |
| Output Value | |
| Output Units | |
| Output Notes | Significant GAPDH silencing observed for R8-PLP 10 mol% at higher lipid:siRNA capacities (e.g., 400 nM GAPDH siRNA at 20:1 w:w ~41% relative expression; 200 nM at 10:1 w:w ~83% relative expression). |
| Toxicity Notes | No significant cytotoxicity detected for R8-PLP 10 mol% vs untreated controls at 200 nM siRNA exposure. |
| Curation Notes |