scholarly journals Multifunctional Nucleus-targeting Nanoparticles with Ultra-high Gene Transfection Efficiency for In Vivo Gene Therapy

Theranostics ◽  
2017 ◽  
Vol 7 (6) ◽  
pp. 1633-1649 ◽  
Author(s):  
Ling Li ◽  
Xia Li ◽  
Yuzhe Wu ◽  
Linjiang Song ◽  
Xi Yang ◽  
...  
2018 ◽  
Vol 9 (1) ◽  
pp. 4-11 ◽  
Author(s):  
Aparna Bansal ◽  
Himanshu

Introduction: Gene therapy has emerged out as a promising therapeutic pave for the treatment of genetic and acquired diseases. Gene transfection into target cells using naked DNA is a simple and safe approach which has been further improved by combining vectors or gene carriers. Both viral and non-viral approaches have achieved a milestone to establish this technique, but non-viral approaches have attained a significant attention because of their favourable properties like less immunotoxicity and biosafety, easy to produce with versatile surface modifications, etc. Literature is rich in evidences which revealed that undoubtedly, non–viral vectors have acquired a unique place in gene therapy but still there are number of challenges which are to be overcome to increase their effectiveness and prove them ideal gene vectors. Conclusion: To date, tissue specific expression, long lasting gene expression system, enhanced gene transfection efficiency has been achieved with improvement in delivery methods using non-viral vectors. This review mainly summarizes the various physical and chemical methods for gene transfer in vitro and in vivo.


2016 ◽  
Vol 4 (8) ◽  
pp. 1233-1243 ◽  
Author(s):  
Chen Xu ◽  
Bingran Yu ◽  
Hao Hu ◽  
Muhammad Naeem Nizam ◽  
Wei Yuan ◽  
...  

A series of effective cationic conjugations of lipid molecules with low cytotoxicity and high gene transfection efficiency were readily designed for gene therapy and tumor treatment.


2021 ◽  
Vol 9 (7) ◽  
pp. 2454-2466
Author(s):  
Yingying Liu ◽  
Yuli Zhou ◽  
Jinfeng Xu ◽  
Hui Luo ◽  
Yao Zhu ◽  
...  

A novel dual-targeted cationic microbubbles help to improve gene transfection efficiency.


2015 ◽  
Vol 6 (5) ◽  
pp. 780-796 ◽  
Author(s):  
Cheng Wang ◽  
Xiuli Bao ◽  
Xuefang Ding ◽  
Yang Ding ◽  
Sarra Abbad ◽  
...  

A novel coating polymer LPHF is developed for the first time to elevate the transfection efficiency of DP binary polyplexes in vitro and in vivo.


Biomaterials ◽  
2014 ◽  
Vol 35 (28) ◽  
pp. 8261-8272 ◽  
Author(s):  
Raviraj Vankayala ◽  
Chi-Shiun Chiang ◽  
Jui-I. Chao ◽  
Chiun-Jye Yuan ◽  
Shyr-Yeu Lin ◽  
...  

PLoS ONE ◽  
2016 ◽  
Vol 11 (12) ◽  
pp. e0166673 ◽  
Author(s):  
Jing Hu ◽  
Manman Zhu ◽  
Kehai Liu ◽  
Hua Fan ◽  
Wenfang Zhao ◽  
...  

2016 ◽  
Vol 4 (5) ◽  
pp. 997-1008 ◽  
Author(s):  
Juan Lv ◽  
Jing Yang ◽  
Xuefang Hao ◽  
Xiangkui Ren ◽  
Yakai Feng ◽  
...  

In recent years, gene therapy has evoked an increasing interest in clinical treatments of coronary diseases because it is a potential strategy to realize rapid endothelialization of artificial vascular grafts.


2016 ◽  
Vol 4 (39) ◽  
pp. 6462-6467 ◽  
Author(s):  
Sheng-Gang Ding ◽  
Lei Yu ◽  
Long-Hai Wang ◽  
Lin-Ding Wang ◽  
Zhi-Qiang Yu ◽  
...  

Polycations have high DNA condensing ability, low immunogenicity, and great adaptability, which make them promising for gene delivery.


2020 ◽  
Author(s):  
Kai Wang ◽  
Fusheng Shang ◽  
Dagui Chen ◽  
Jianpeng Jiao ◽  
Tieliu Cao ◽  
...  

Abstract The development of highly efficient non-viral gene vector systems has very important application value in the field of cancer therapy. The high protein content of proteolipids allows for high biocompatibility, low immunogenicity, and surface modification of proteins to confer more targeted drug/gene function. For the first time, this study selected transferrin, which has hepatocellular carcinoma cell targeting function, with a liposome backbone material to construct transferrin liposome (Tf-PL), and load acetylcholinesterase (AChE) therapeutic gene for in vitro and in vivo functions evaluation. The results showed that the Tf-PL transfection efficiency was higher than that of commercial Lipo 2000, low cytotoxicity and targeted ability to liver cancer SMMC-7721 cells. After tail vein injection, Tf-PL/AChE can effectively target to liver cancer, significantly inhibiting the growth of liver cancer xenografts in nude mice, prolonging the survival time of tumor-bearing nude mice, and also does not cause significant systemic toxicities. Our study provides a strategy for proteolipids targeting the transferrin receptor to carry therapeutic gene therapy for tumors. This method has strong tumor affinity and can provide an effective vector selection for precise tumor therapy.


2019 ◽  
Author(s):  
Sofia Bisso ◽  
Simona Mura ◽  
Bastien Castagner ◽  
Patrick Couvreur ◽  
Jean-Christophe Leroux

AbstractDespite many years of research and a few success stories with gene therapeutics, efficient and safe DNA delivery remains a major bottleneck for the clinical translation of gene-based therapies. Gene transfection with calcium phosphate (CaP) nanoparticles brings the advantages of low toxicity, high DNA entrapment efficiency and good endosomal escape properties. The macroscale aggregation of CaP nanoparticles can be easily prevented through surface coating with bisphosphonate conjugates. Bisphosphonates, such as alendronate, recently showed promising anticancer effects. However, their poor cellular permeability and preferential bone accumulation hamper their full application in chemotherapy. Here, we investigated the dual delivery of plasmid DNA and alendronate using CaP nanoparticles, with the goal to facilitate cellular internalization of both compounds and potentially achieve a combined pharmacological effect on the same or different cell lines. A pH-sensitive poly(ethylene glycol)-alendronate conjugate was synthetized and used to formulate stable plasmid DNA-loaded CaP nanoparticles. These particles displayed good transfection efficiency in cancer cells and a strong cytotoxic effect on macrophages. The in vivo transfection efficiency, however, remained low, calling for an improvement of the system, possibly with respect to the extent of particle uptake and their physical stability.Graphical abstract


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