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反义寡核苷酸应用于脑部疾病治疗的研究进展
引用本文:李云凤,孟艳秋,徐亮.反义寡核苷酸应用于脑部疾病治疗的研究进展[J].国际药学研究杂志,2017,44(8).
作者姓名:李云凤  孟艳秋  徐亮
作者单位:1. 110142 沈阳,沈阳化工大学;100850 北京,军事医学科学院毒物药物研究所,抗毒药物与毒理学国家重点实验室;2. 沈阳化工大学, 沈阳,110142;3. 100850 北京,军事医学科学院毒物药物研究所,抗毒药物与毒理学国家重点实验室
基金项目:国家自然科学基金资助项目,北京市科技新星计划资助项目
摘    要:随着生物技术的快速发展,反义核酸药物倍受关注.反义寡核苷酸(ASON)通过作用于疾病相关靶mRNA,干扰基因表达发挥治疗作用,具有特异性高、毒副作用低等特点.基于脑靶向载体技术和化学修饰,ASON在脑部疾病的治疗领域显示了广阔前景.本文综述了ASON药物的研发现状,及其应用于阿尔茨海默病、帕金森病及脑肿瘤等常见脑部疾病治疗的研究进展.

关 键 词:反义寡核苷酸  阿尔茨海默病  帕金森病  脑肿瘤

Antisense oligonucleotide in the treatment of brain diseases:research advances
LI Yun-feng,MENG Yan-qiu,XU Liang.Antisense oligonucleotide in the treatment of brain diseases:research advances[J].Foreign Medical Sciences(Section of Pharmarcy),2017,44(8).
Authors:LI Yun-feng  MENG Yan-qiu  XU Liang
Abstract:Antisense oligonucleotide(ASON)has attracted a growing attention with the rapid development of biological tech?nology. ASON can be designed to target mRNA in a sequence-specific manner to stop,alter,or induce particular disease-related gene functions. Thus,its drugs have emerged as a very promising new class of therapeutics at the molecular genetic level with high specifici?ty and low toxicity. ASON has shown good application prospects in the treatment of brain diseases,by means of brain-targeted delivery and chemical modification. In this review,we summarize the latest development of ASON drugs and their applications on the treatment of Alzheimer disease,Parkinson′s disease and brain tumors.
Keywords:antisense oligonucleotide  Alzheimers disease  Parkinson′s disease  brain tumors
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