Pharmacokinetics of Viral and Non-Viral Gene Delivery Vectors

Author(s):  
Martin Meyer ◽  
Gururaj Rao ◽  
Ke Ren ◽  
Jeffrey Hughes
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

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.


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