Strategies and materials of SMARTnon-viral vectors: Overcoming the barriers for brain gene therapy

doi:10.1016/j.nantod.2020.101006

	Strategies and materials of SMARTnon-viral vectors: Overcoming the barriers for brain gene therapy
	Li, Yan 1,2; Liu, Linying 1,2; Ji, Weihong 1,2; Peng, Huang 1,2; Zhao, Ruichen 1,2; Zhang, Xin 1,2
刊名	NANO TODAY
	2020-12-01
卷号	35 页码:22
关键词	Brain gene therapy Non-viral vectors Blood-brain barrier Endosome trapping Real-time non-invasive monitoring
ISSN号	1748-0132
DOI	10.1016/j.nantod.2020.101006
英文摘要	Most brain diseases such as tumors and neurodegenerative diseases are devastating for patients with high morbidity and mortality. Gene therapy is a promising strategy for the treatment of brain diseases. The widespread use of brain gene therapy requires the development of safe and effective non-viral vectors as its efficiency is seriously limited due to the instability, unfavorable biodistribution, and poor cellular uptake of genetic drugs. It is required that non-viral vectors should deliver the genetic drugs into brain and further into their action site of specific cells to enhance the therapeutic efficacy and minimize the nonspecific toxicity. However, it poses major challenges to non-viral vectors as there are many transport hurdles existing for brain gene therapy. These tissue and cellular hurdles mainly include blood-brain barrier, nonspecific cellular uptake, endosome trapping, and invasive monitoring. In this review, it provides an overview of the non-viral vectors used for brain gene therapy, and discusses the strategies and materials that have been developed for SMARTnon-viral vectors to overcome these barriers for brain gene therapy. The review discusses the potential of translation for SMARTnon-viral vectors to the clinic. Furthermore, the review provides an outlook for this field. (C) 2020 Elsevier Ltd. All rights reserved.
资助项目	National High Technology Research and Development Program[2016YFA0200303] ; National Natural Science Foundation of China[21905283] ; National Natural Science Foundation of China[31771095] ; National Natural Science Foundation of China[21875254] ; Beijing Natural Science Foundation[L172046] ; Beijing Natural Science Foundation[2192057]
WOS关键词	CONVECTION-ENHANCED DELIVERY ; EXOSOME-MEDIATED DELIVERY ; IRON-OXIDE NANOPARTICLES ; SMALL INTERFERING RNA ; SIRNA DELIVERY ; ALZHEIMERS-DISEASE ; IN-VIVO ; FOCUSED ULTRASOUND ; PARKINSONS-DISEASE ; DRUG-DELIVERY
WOS研究方向	Chemistry ; Science & Technology - Other Topics ; Materials Science
语种	英语
出版者	ELSEVIER SCI LTD
WOS记录号	WOS:000600849700062
资助机构	National High Technology Research and Development Program ; National Natural Science Foundation of China ; Beijing Natural Science Foundation
内容类型	期刊论文
源URL	[http://ir.ipe.ac.cn/handle/122111/43109]
专题	中国科学院过程工程研究所
通讯作者	Zhang, Xin
作者单位	1.Chinese Acad Sci, Inst Proc Engn, State Key Lab Biochem Engn, Beijing 100190, Peoples R China 2.Univ Chinese Acad Sci, Beijing 100049, Peoples R China
推荐引用方式 GB/T 7714	Li, Yan,Liu, Linying,Ji, Weihong,et al. Strategies and materials of SMARTnon-viral vectors: Overcoming the barriers for brain gene therapy[J]. NANO TODAY,2020,35:22.
APA	Li, Yan,Liu, Linying,Ji, Weihong,Peng, Huang,Zhao, Ruichen,&Zhang, Xin.(2020).Strategies and materials of SMARTnon-viral vectors: Overcoming the barriers for brain gene therapy.NANO TODAY,35,22.
MLA	Li, Yan,et al."Strategies and materials of SMARTnon-viral vectors: Overcoming the barriers for brain gene therapy".NANO TODAY 35(2020):22.