Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disorders

被引：36

作者：

Gallego, Idoia ^{[1
,2
]}

Villate-Beitia, Ilia ^{[1
,3
]}

Martinez-Navarrete, Gema ^{[3
,4
]}

Menendez, Margarita ^{[5
,6
]}

Lopez-Mendez, Tania ^{[1
,3
]}

Soto-Sanchez, Cristina ^{[3
,4
]}

Zarate, Jon ^{[1
,3
]}

Puras, Gustavo ^{[1
,3
]}

Fernandez, Eduardo ^{[3
,4
]}

Luis Pedraz, Jose ^{[1
,3
]}

机构：

[1] Univ Basque Country, UPV EHU, NanoBioCel Grp, Vitoria, Spain

[2] Basque Fdn Sci, Ikerbasque, Bilbao, Spain

[3] Biomed Res Networking Ctr Bioengn Biomat & Nanome, Malaga, Spain

[4] Miguel Hernandez Univ, Neuroprothesis & Neuroengn Res Grp, Elche, Spain

[5] Super Council Sci Invest IQFR CSIC, Rocasolano Phys Chem Inst, Madrid, Spain

[6] Biomed Res Networking Ctr Resp Dis CIBERES, Madrid, Spain

来源：

NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE | 2019年 / 17卷

关键词：

Non-viral vector; Niosomes; Minicircle Transfection; Gene therapy; Retina; TRANSFECTION EFFICIENCY; HELPER LIPIDS; THERAPY; NANOPARTICLES; FORMULATION; LIPOPLEXES; PROGRESS; CHARGE; SIZE;

D O I：

10.1016/j.nano.2018.12.018

中图分类号：

TB3 [工程材料学];

学科分类号：

0805 ; 080502 ;

摘要：

Low transfection efficiency is a major challenge to overcome in non-viral approaches to reach clinical practice. Our aim was to explore new strategies to achieve more efficient non-viral gene therapies for clinical applications and in particular, for retinal diseases. Cationic niosomes and three GFP-encoding genetic materials consisting on minicircle (2.3 kb), its parental plasmid (3.5 kb) and a larger plasmid (5.5 kb) were combined to fonn nioplexes. Once fully physicochemically characterized, in vitro experiments in ARPE-19 retina epithelial cells showed that transfection efficiency of minicircle nioplexes doubled that of plasmids ones, maintaining good cell viability in all cases. Transfections in retinal primary cells and injections of nioplexes in rat retinas confirmed the higher capacity of cationic niosomes vectoring minicircle to deliver the genetic material into retina cells. Therefore, nioplexes based on cationic niosomes vectoring minicircle DNA represent a potential tool for the treatment of inherited retinal diseases. (C) 2019 Elsevier Inc. All rights reserved.

引用

页码：308 / 318

页数：11

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