Non-viral vectors for gene-based therapy

被引：2514

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：

美国国家卫生研究院; 美国国家科学基金会;

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 50 条

[1] Non-viral vectors for gene-based therapy
Hao Yin
Rosemary L. Kanasty
Ahmed A. Eltoukhy
Arturo J. Vegas
J. Robert Dorkin
Daniel G. Anderson
Nature Reviews Genetics, 2014, 15 : 541 - 555
[2] Genocompatibility of non-viral vectors for gene-based therapies
Akhtar, S
Omidi, Y
Hollins, A
Drayton, R
Benboubetra, M
JOURNAL OF GENE MEDICINE, 2004, 6 (09): : S5 - S5
[3] Non-viral and viral vectors for gene therapy
Boulaiz, H
Marchal, JA
Prados, J
Melguizo, C
Aránega, A
CELLULAR AND MOLECULAR BIOLOGY, 2005, 51 (01) : 3 - 22
[4] Viral and non-viral vectors for cancer gene therapy
Cristiano, RJ
ANTICANCER RESEARCH, 1998, 18 (5A) : 3241 - 3245
[5] Phosphonolipids as non-viral vectors for gene therapy
Floch, V
Le Bolc'h, G
Gable-Guillaume, C
Le Bris, N
Yaouanc, JJ
des Abbayes, H
Férec, C
Clément, JC
EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY, 1998, 33 (12) : 923 - 934
[6] Endocytosis in gene therapy with non-viral vectors
de Garibay, Aritz Perez Ruiz
WIENER MEDIZINISCHE WOCHENSCHRIFT, 2016, 166 (7-8) : 227 - 235
[7] Polymer-based non-viral vectors for gene therapy in the skin
Tortajada, Luz
Felip-Leon, Carles
Vicent, Maria J.
POLYMER CHEMISTRY, 2022, 13 (06) : 718 - 735
[8] Internalization Pathways of Non-Viral Vectors for Gene Therapy
Anghel, Ersilia
Shi, Julie
Pun, Suzie
FASEB JOURNAL, 2012, 26
[9] Design of modular non-viral gene therapy vectors
De Laporte, L
Rea, JC
Shea, LD
BIOMATERIALS, 2006, 27 (07) : 947 - 954
[10] Non-viral, coated nanoparticles as vectors for gene therapy
Campbell, Fred
Welser, Katharina
Mohammadi, Atefeh
Kudsiova, Laila
Lawrence, Margaret J.
Tabor, Alethea B.
Hailes, Helen C.
ABSTRACTS OF PAPERS OF THE AMERICAN CHEMICAL SOCIETY, 2011, 242

← 1 2 3 4 5 →