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Strategies and materials of "SMART" non-viral vectors: Overcoming the barriers for brain gene therapy
Nano Today ( IF 17.4 ) Pub Date : 2020-12-01 , DOI: 10.1016/j.nantod.2020.101006
Yan Li , Linying Liu , Weihong Ji , Huang Peng , Ruichen Zhao , Xin Zhang

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 "SMART" non-viral vectors to overcome these barriers for brain gene therapy. The review discusses the potential of translation for "SMART" non-viral vectors to the clinic. Furthermore, the review provides an outlook for this field.

中文翻译:

“SMART”非病毒载体的策略与材料:克服脑基因治疗的障碍

摘要 大多数脑部疾病如肿瘤和神经退行性疾病对高发病率和高死亡率的患者来说是毁灭性的。基因疗法是治疗脑部疾病的一种有前景的策略。脑基因治疗的广泛应用需要开发安全有效的非病毒载体,因为基因药物的不稳定性、不利的生物分布和细胞吸收不良,严重限制了其效率。需要非病毒载体将基因药物输送到大脑中,并进一步进入其特定细胞的作用部位,以提高治疗效果并最大限度地减少非特异性毒性。然而,它对非病毒载体构成了重大挑战,因为脑基因治疗存在许多运输障碍。这些组织和细胞障碍主要包括血脑屏障、非特异性细胞摄取、内体捕获和侵入性监测。在这篇综述中,它概述了用于脑基因治疗的非病毒载体,并讨论了为“SMART”非病毒载体开发的策略和材料,以克服脑基因治疗的这些障碍。该评论讨论了将“SMART”非病毒载体转化为临床的潜力。此外,该评论提供了该领域的前景。该评论讨论了将“SMART”非病毒载体转化为临床的潜力。此外,该评论提供了该领域的前景。该评论讨论了将“SMART”非病毒载体转化为临床的潜力。此外,该评论提供了该领域的前景。
更新日期:2020-12-01
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