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| Strategies and materials of SMARTnon-viral vectors: Overcoming the barriers for brain gene therapy | |
| Li, Yan1,2; Liu, Linying1,2; Ji, Weihong1,2; Peng, Huang1,2; Zhao, Ruichen1,2; Zhang, Xin1,2 | |
| 2020-12-01 | |
| Source Publication | NANO TODAY
 |
| ISSN | 1748-0132 |
| Volume | 35Pages:22 |
| Abstract | 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. |
| Keyword | Brain gene therapy Non-viral vectors Blood-brain barrier Endosome trapping Real-time non-invasive monitoring |
| DOI | 10.1016/j.nantod.2020.101006 |
| Language | 英语 |
| WOS Keyword | CONVECTION-ENHANCED DELIVERY ; EXOSOME-MEDIATED DELIVERY ; IRON-OXIDE NANOPARTICLES ; SMALL INTERFERING RNA ; SIRNA DELIVERY ; ALZHEIMERS-DISEASE ; IN-VIVO ; FOCUSED ULTRASOUND ; PARKINSONS-DISEASE ; DRUG-DELIVERY |
| Funding Project | 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 Research Area | Chemistry ; Science & Technology - Other Topics ; Materials Science |
| WOS Subject | Chemistry, Multidisciplinary ; Nanoscience & Nanotechnology ; Materials Science, Multidisciplinary |
| Funding Organization | National High Technology Research and Development Program ; National Natural Science Foundation of China ; Beijing Natural Science Foundation |
| WOS ID | WOS:000600849700062 |
| Publisher | ELSEVIER SCI LTD |
| Citation statistics | |
| Document Type | 期刊论文 |
| Identifier | http://ir.ipe.ac.cn/handle/122111/43110 |
| Collection | 中国科学院过程工程研究所 |
| Corresponding Author | Zhang, Xin |
| Affiliation | 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 |
| Recommended Citation 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. |
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