Non-viral vectors for gene-based therapy

被引：2600

作者：

Yin, Hao ^{[1
]}

Kanasty, Rosemary L. ^{[1
,2
]}

Eltoukhy, Ahmed A. ^{[1
]}

Vegas, Arturo J. ^{[1
,3
]}

Dorkin, J. Robert ^{[1
,4
]}

Anderson, Daniel G. ^{[1
,2
,3
,5
,6
]}

机构：

[1] MIT, David H Koch Inst Integrat Canc Res, Cambridge, MA 02142 USA

[2] MIT, Dept Chem Engn, Cambridge, MA 02142 USA

[3] Childrens Hosp, Dept Anesthesiol, Boston, MA 02115 USA

[4] MIT, Dept Biol, Cambridge, MA 02142 USA

[5] MIT, Harvard Mit Div Hlth Sci & Technol, Cambridge, MA 02139 USA

[6] MIT, Inst Med Engn & Sci, Cambridge, MA 02142 USA

来源：

NATURE REVIEWS GENETICS | 2014年 / 15卷 / 08期

基金：

美国国家科学基金会; 美国国家卫生研究院;

关键词：

IN-VIVO DELIVERY; PLASMID-LIPID PARTICLES; MODIFIED MESSENGER-RNA; TRANSGENE EXPRESSION; TARGETED DELIVERY; SLEEPING-BEAUTY; VIRAL VECTORS; ADMINISTERED SIRNA; NONHUMAN-PRIMATES; SYSTEMIC DELIVERY;

D O I：

10.1038/nrg3763

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Gene-based therapy is the intentional modulation of gene expression in specific cells to treat pathological conditions. This modulation is accomplished by introducing exogenous nucleic acids such as DNA, mRNA, small interfering RNA (siRNA), microRNA (miRNA) or antisense oligonucleotides. Given the large size and the negative charge of these macromolecules, their delivery is typically mediated by carriers or vectors. In this Review, we introduce the biological barriers to gene delivery in vivo and discuss recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems, some of which are currently undergoing testing in clinical trials. The diversity of these systems highlights the recent progress of gene-based therapy using non-viral approaches.

引用

页码：541 / 555

页数：15

共 159 条

[61]

Kanasty R, 2013, NAT MATER, V12, P967, DOI [10.1038/NMAT3765, 10.1038/nmat3765]

[62] Action and Reaction: The Biological Response to siRNA and Its Delivery Vehicles [J].

Kanasty, Rosemary L. ;

Whitehead, Kathryn A. ;

Vegas, Arturo J. ;

Anderson, Daniel G. .

MOLECULAR THERAPY, 2012, 20 (03) :513-524

[63] Suppression of RNA recognition by Toll-like receptors:: The impact of nucleoside modification and the evolutionary origin of RNA [J].

Karikó, K ;

Buckstein, M ;

Ni, HP ;

Weissman, D .

IMMUNITY, 2005, 23 (02) :165-175

[64] Increased Erythropoiesis in Mice Injected With Submicrogram Quantities of Pseudouridine-containing mRNA Encoding Erythropoietin [J].

Kariko, Katalin ;

Muramatsu, Hiromi ;

Keller, Jason M. ;

Weissman, Drew .

MOLECULAR THERAPY, 2012, 20 (05) :948-953

[65] THE FATE OF PLASMID DNA AFTER INTRAVENOUS-INJECTION IN MICE - INVOLVEMENT OF SCAVENGER RECEPTORS IN ITS HEPATIC-UPTAKE [J].

KAWABATA, K ;

TAKAKURA, Y ;

HASHIDA, M .

PHARMACEUTICAL RESEARCH, 1995, 12 (06) :825-830

[66] State-of-the-art gene-based therapies: the road ahead [J].

Kay, Mark A. .

NATURE REVIEWS GENETICS, 2011, 12 (05) :316-328

[67] A robust system for production of minicircle DNA vectors [J].

Kay, Mark A. ;

He, Cheng-Yi ;

Chen, Zhi-Ying .

NATURE BIOTECHNOLOGY, 2010, 28 (12) :1287-U96

[68]

Kim Sung Wan, 2012, Cold Spring Harb Protoc, V2012, P433, DOI 10.1101/pdb.ip068619

[69] Design and gene delivery activity of modified polyethylenimines [J].

Kircheis, R ;

Wightman, L ;

Wagner, E .

ADVANCED DRUG DELIVERY REVIEWS, 2001, 53 (03) :341-358

[70] An inverted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery [J].

Koltover, I ;

Salditt, T ;

Rädler, JO ;

Safinya, CR .

SCIENCE, 1998, 281 (5373) :78-81

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