Trimethylated chitosans as non-viral gene delivery vectors: Cytotoxicity and transfection efficiency

Author(s):  
T KEAN ◽  
S ROTH ◽  
M THANOU
Molecules ◽  
2020 ◽  
Vol 25 (4) ◽  
pp. 975 ◽  
Author(s):  
Xiao-Ru Wu ◽  
Ji Zhang ◽  
Ju-Hui Zhang ◽  
Ya-Ping Xiao ◽  
Xi He ◽  
...  

The construction of efficient and low toxic non-viral gene delivery vectors is of great significance for gene therapy. Herein, two novel polycations were constructed via Michael addition from low molecular weight polyethylenimine (PEI) 600 Da and amino acid-containing linkages. Lysine and histidine were introduced for the purpose of improved DNA binding and pH buffering capacity, respectively. The ester bonds afforded the polymer biodegradability, which was confirmed by the gel permeation chromatography (GPC) measurement. The polymers could well condense DNA into nanoparticles and protect DNA from degradation by nuclease. Compared with PEI 25 kDa, these polymers showed higher transfection efficiency, lower toxicity, and better serum tolerance. Study of this mechanism revealed that the polyplexes enter the cells mainly through caveolae-mediated endocytosis pathway; this, together with their biodegradability, facilitates the internalization of polyplexes and the release of DNA. The results reveal that the amino acid-linked low molecular weight PEI polymers could serve as promising candidates for non-viral gene delivery.


2020 ◽  
Vol 20 ◽  
Author(s):  
L. Hajba ◽  
A. Guttman

: Adeno-associated virus (AAV) is one of the most promising viral gene delivery vectors with long-term gene expression and disease correction featuring high efficiency and excellent safety in human clinical trials. During the production of AAV vectors,there are several quality control (QC)parameters that should be rigorously monitored to comply with clini-cal safety and efficacy. This review gives a short summary of the most frequently used AVV production and purification methods,focusing on the analytical techniques applied to determine the full/empty capsid ratio and the integrity of the encapsidated therapeutic DNA of the products.


RSC Advances ◽  
2017 ◽  
Vol 7 (30) ◽  
pp. 18681-18689 ◽  
Author(s):  
De-Chun Chang ◽  
Yi-Mei Zhang ◽  
Ji Zhang ◽  
Yan-Hong Liu ◽  
Xiao-Qi Yu

The structure–activity relationships of cyclen-based cationic lipids as non-viral gene delivery vectors were studied and clarified.


2016 ◽  
Vol 26 (10) ◽  
pp. 2401-2407 ◽  
Author(s):  
Jia Ju ◽  
Meng-Lei Huan ◽  
Ning Wan ◽  
Yi-Lin Hou ◽  
Xi-Xi Ma ◽  
...  

2013 ◽  
Vol 82 (4) ◽  
pp. 376-383 ◽  
Author(s):  
Qiang Liu ◽  
Wen-Jing Yi ◽  
Yi-Mei Zhang ◽  
Ji Zhang ◽  
Liandi Guo ◽  
...  

2018 ◽  
Vol 27 (1) ◽  
pp. 1-11 ◽  
Author(s):  
Shuang Fu ◽  
Xiaodong Xu ◽  
Yu Ma ◽  
Shubiao Zhang ◽  
Shufen Zhang

Author(s):  
Ilona Uzieliene ◽  
Ursule Kalvaityte ◽  
Eiva Bernotiene ◽  
Ali Mobasheri

Strategies for delivering nucleic acids into damaged and diseased tissues have been divided into two major areas: viral and non-viral gene therapy. In this mini-review article we discuss the application of gene therapy for the treatment of osteoarthritis (OA), one of the most common forms of arthritis. We focus primarily on non-viral gene therapy and cell therapy. We briefly discuss the advantages and disadvantages of viral and non-viral gene therapy and review the nucleic acid transfer systems that have been used for gene delivery into articular chondrocytes in cartilage from the synovial joint. Although viral gene delivery has been more popular due to its reported efficiency, significant effort has gone into enhancing the transfection efficiency of non-viral delivery, making non-viral approaches promising tools for further application in basic, translational and clinical studies on OA. Non-viral gene delivery technologies have the potential to transform the future development of disease-modifying therapeutics for OA and related osteoarticular disorders. However, further research is needed to optimize transfection efficiency, longevity and duration of gene expression.


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