Strategies and materials of SMARTnon-viral vectors: Overcoming the barriers for brain gene therapy
文献类型：期刊论文
| 作者 | Li, Yan1,2; Liu, Linying1,2; Ji, Weihong1,2; Peng, Huang1,2; Zhao, Ruichen1,2; Zhang, Xin1,2 |
| 刊名 | NANO TODAY
 |
| 出版日期 | 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. |
| 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 |
| 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研究方向 | Chemistry ; Science & Technology - Other Topics ; Materials Science |
| 语种 | 英语 |
| WOS记录号 | WOS:000600849700062 |
| 出版者 | ELSEVIER SCI LTD |
| 资助机构 | National High Technology Research and Development Program ; National Natural Science Foundation of China ; Beijing Natural Science Foundation |
| 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. |
入库方式： OAI收割
来源：过程工程研究所
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