Developing a novel cholesterol-based nanocarrier with high transfection efficiency and serum compatibility for gene therapy
Journal
Journal of the Formosan Medical Association
Journal Volume
118
Journal Issue
4
Pages
766-775
Date Issued
2019
Author(s)
Abstract
BACKGROUND/PURPOSE: Primary cells are sensitive to culture conditions, which can be more difficult to get efficient transfection. The purpose of this study is to develop a serum-compatible cholesterol-based nanocarrier for delivering therapeutic nucleic acids into cells efficiently for future clinical gene therapy. METHODS: nanocarrier. Transfection efficiency and cytotoxicity in serum and non-serum conditions were evaluated. Florescent-labeled oligonucleotides (ODNs) were transfected as indicators. Fluorescent microscopy, confocal microscopy, and flow cytometry analysis were used for evaluations. Besides, we also delivered functional antisense c-myc ODNs as surrogates for specific gene manipulation in vitro. RESULTS: nanocarrier had exceeded 90% transfection efficiency, which was much better than common commercialized transfection reagents under same conditions. Such nanocarrier not only worked well in cell lines, but also ideal for gene delivery in primary cells. CONCLUSION: With high transfection efficiency and serum compatibility, this novel biocompatible cholesterol-based nanocarrier provides an ideal platform especially for RNAi-based gene manipulation. It also opens a wide range of biomedical applications for in vivo cell tracking and gene therapeutics for clinical usage.
SDGs
Publisher
Elsevier B.V.
Type
journal article