中国组织工程研究

中国组织工程研究 ›› 2010, Vol. 14 ›› Issue (37): 7004-7007.doi: 10.3969/j.issn.1673-8225.2010.37.039

• 组织构建学术探讨 tissue construction academic discussion • 上一篇    下一篇

小干扰RNA在哺乳动物脑组织中的递送

张  奇，张  琳，颜  荣，杨新宇   

  1. 天津医科大学总医院神经外科，天津市神经病学研究所，天津市神经损伤变异与再生重点实验室，天津市  300052　
  • 出版日期:2010-09-10 发布日期:2010-09-10
  • 通讯作者: 杨新宇，博士，教授，主任医师，硕士生导师，天津医科大学总医院神经外科，天津市神经病学研究所，天津市神经损伤变异与再生重点实验室，天津市300052 paperxyyang@gmail.com
  • 作者简介:张奇★，男，1985年生，山东省安丘市人，汉族，天津医科大学在读硕士，主要从事脑创伤和脑血管病的外科治疗研究。 Dake413@163.com
  • 基金资助:

    国家自然基金资助项目(30973090)；天津市自然科学基金资助项目(09JCZDJC17200)；天津市高等学校科技发展基金计划项目(20070202)。

Delivery of small-interfering RNA to the mammalian brain

Zhang Qi, Zhang Lin, Yan Rong, Yang Xin-yu   

  1. Department of Neurosurgery, General Hospital of Tianjin Medical University, Tianjin Key Laboratory of Injuries, Variations and Regeneration of Nervous System, Tianjin Neurological Institute, Tianjin  300052, China
  • Online:2010-09-10 Published:2010-09-10
  • Contact: Yang Xin-yu, Doctor, Professor, Chief physician, Master’s supervisor, Department of Neurosurgery, General Hospital of Tianjin Medical University, Tianjin Key Laboratory of Injuries, Variations and Regeneration of Nervous System, Tianjin Neurological Institute, Tianjin 300052, China paperxyyang@gmail.com
  • About author:Zhang Qi★, Studying for master’s degree, Department of Neurosurgery, General Hospital of Tianjin Medical University, Tianjin Key Laboratory of Injuries, Variations and Regeneration of Nervous System, Tianjin Neurological Institute, Tianjin 300052, China Dake413@163.com
  • Supported by:

    the National Natural Science Foundation of China, No. 30973090*; Natural Science Foundation of Tianjin, No. 09JCZDJC17200*; High School Science & Technology Fund Planning Project of Tianjin, No. 20070202*

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306

被引次数

1

摘要：

背景：作为一种能够下调基因表达的新的基因治疗方法，RNA干扰在神经科学疾病的治疗中有很大的潜能。通过引入与内源靶基因具有相同序列的小干扰RNA，可以人为地诱导序列特异性的mRNA降解，达到阻止该基因表达的目的。
目的：针对RNA干扰在神经科学疾病治疗方面的应用，探讨小干扰RNA在哺乳动物脑组织中的递送情况。
方法：以“siRNA，delivery，brain”和“siRNA, delivery，vivo”为检索词，计算机检索PubMed home，按纳入和排除标准对文献进行筛选和质量评价，共纳入27篇文章。从小干扰RNA合成、脑组织中的递送及其抑制效率和不良反应3方面进行总结。
结果与结论：近来RNA干扰研究中，高精度预测小干扰RNA的方法能合成出高特异性的小干扰RNA；小干扰RNA在脑组织中的递送主要采用局部注射的方式，并且在递送载体的帮助下，抑制效率在不断地提高；随着RNA干扰技术研究的深入，其不良反应也逐渐受到人们重视。总之，作为一种新的治疗策略，RNA干扰在神经科学疾病的治疗中有着很大的潜能，并可能给药物研发带来新的变革。

关键词: 小干扰RNA, RNA干扰, 脑组织, 递送, 不良反应

Abstract:

BACKGROUND: As a new method of gene therapy, small-interfering RNA (RNAi) can reduce the expression of target gene, which has a great potential in the treatment of neuroscience diseases. By introducing siRNA with the same sequence of endogenous target gene, the technology can artificially induce sequence-specific mRNA degradation to prevent the gene expression.
OBJECTIVE: To explore the delivery of siRNA to the mammalian brain in the treatment of neuroscience diseases.
METHODS: The database of PubMed home was retrieved with key words of “siRNA, delivery, brain” and “siRNA, delivery, vivo” by computer, separately. The literature was selected according to inclusion and exclusion criteria. Totally 27 papers were analyzed from the synthesis of siRNA, delivery in the brain, its inhibition efficiency and side effects.
RESULTS AND CONCLUSION: In recent RNAi studies, high-precision prediction method of siRNA could be used to synthesize highly specific siRNA; siRNA might be delivered to the mammalian brain, mainly through local injection approach, and with the help of the delivery carrier, the inhibition efficiency was constantly improved; with the study of RNAi technology, its side effects was also gaining the attention of researchers. In brief, as a novel treatment strategy, RNAi has a great potential in the treatment of neuroscience diseases and can bring a new revolution in drug discovery and development.

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