Nucleic acid drugs have emerged as important therapeutics but their clinical application has been greatly limited by their large molecular weight, high polarity, negative charge and short half-life. Cationic liposomes (CLs) have gained wide attention as non-viral vectors for nucleic acid delivery. However, the absolute transfection efficiency of CLs can still be enhanced while their cytotoxicity should be decreased simultaneously. Ginsenosides, obtained from natural plants, possess a similar steroid structure to cholesterol and might be an alternative to cholesterol for acting as a membrane stabilizer of CLs. Herein, seven kinds of ginsenoside-based compounds were utilized to prepare CLs (GCLs) and their efficacy in siRNA delivery was investigated. The particle sizes of the GCLs were 90-300 nm and the siRNA delivery efficiencies were in the range of 23.6%-78.4%. Rg5-based CLs (Rg5-CLs) exhibited the highest transfection efficiency of 81% and the lowest toxicity, with 82% cell viability obtained even at high concentrations. Ginsenosides are shown as promising biomaterials as membrane stabilizers of CLs. Rg5-CLs have been demonstrated as efficient non-viral vectors with high transfection efficiency and good biocompatibility for gene delivery, possessing great potential for gene therapy.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1039/d1bm01353j | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
A critical view of silk fibroin for non-viral gene therapy.
Int J Biol Macromol
January 2025
National Engineering Laboratory for Modern Silk, College of Textile and Clothing Engineering, Soochow University, No. 199 Ren'ai Road, Industrial Park, Suzhou 215123, PR China. Electronic address:
Exogenous genes are inserted into target cells during gene therapy in order to compensate or rectify disorders brought on by faulty or aberrant genes. However, gene therapy is still in its early stages because of its unsatisfactory therapeutic effects which are mainly due to low transfection efficiency of vectors, high toxicity, and poor target specificity. A natural polymer with numerous bioactive sites, good mechanical qualities, biodegradability, biocompatibility, and processability called silk fibroin has gained attention as a possible gene therapy vector.
View Article and Find Full Text PDFIn this study, we have designed and developed a cationic bolaform C12-(2,3-dihydroxy-N, N-dimethyl-N-(2-ureidoethyl)propan-1-aminium chloride)2 (C12(DDUPAC)2) that is derived from biocompatible molecules. The bolaform C12(DDUPAC)2 has hydroxyl (OH) functionality at both the cationic head groups. The impact of head group structure on the self-assembly and effectiveness of gene transfection and antimicrobial activity was investigated and compared with that of the hydrochloride salt C12-(N, N-dimethyl-N-(2-ureidoethan-1-aminium chloride)2 (C12(DUAC)2) of its precursor molecule.
View Article and Find Full Text PDFSenkyunolide A attenuates cerebral ischemia-reperfusion injury by inhibiting NLRP3-mediated ferroptosis in PC12 cells.
Gen Physiol Biophys
January 2025
Shanghai University of Traditional Chinese Medicine, Shenzhen Hospital, Shenzhen, Guangdong, China.
Cerebral ischemia-reperfusion (I/R) is a serious complication in patients with ischemic stroke. Senkyunolide A (SenA) can alleviate neuronal cell damage induced by cerebral I/R; however, the exact action mechanism remains unclear. An in vitro cellular injury model was established by inducing PC-12 cells with OGD/R.
View Article and Find Full Text PDFMacrophage-specific in vivo RNA editing promotes phagocytosis and antitumor immunity in mice.
Sci Transl Med
January 2025
College of Pharmaceutical Sciences, State Key Laboratory of Advanced Drug Delivery and Release Systems, Zhejiang University, Hangzhou 310058, China.
Macrophages play a central role in antitumor immunity, making them an attractive target for gene therapy strategies. However, macrophages are difficult to transfect because of nucleic acid sensors that can trigger the degradation of foreign plasmid DNA. Here, we developed a macrophage-specific editing (MAGE) system by which compact plasmid DNA encoding a CasRx editor can be delivered to macrophages by a poly(β-amino ester) (PBAE) carrier to bypass the DNA sensor and enable RNA editing in vitro and in vivo.
View Article and Find Full Text PDFDevelopment of Lentiviral Packaging Cells and Scale Up of Production to Meet the Growing Demand in Cell and Gene Therapy.
Curr Gene Ther
January 2025
Research Group Medical Biotechnology & Bioengineering, TH Köln - University of Applied Sciences, Leverkusen, Germany.
Gamma-Retroviral (RVVs) and lentiviral vectors (LVVs) represent indispensable tools in somatic gene therapy, mediating the efficient, stable transfer of therapeutic genes into a variety of human target cells. LVVs, in contrast to RVVs, are capable of stably genetically modifying non-proliferating target cells, making them the superior instrument in cell and gene therapy. To date, the LVV manufacturing process employs human embryonic kidney cells (HEK293) and derivatives thereof transiently transfected with multiple plasmids encoding the required viral vector components.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!