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科学通报  2014 

中国药物研发的新机遇:基于医药大数据的系统性药物重定位

DOI: 10.1360/972013-731, PP. 1790-1796

Keywords: 医药大数据,药物重定位,旧药新用,转化医学,新药研发,计算生物学,生物信息学

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Abstract:

在科研资源相对有限的前提下,如何提高药物研发的投入产出比,最终为全社会提供安全、有效和廉价的药品,是一个对我国生物医药基础和应用研究均具有重大意义的问题.面对国际上原创新药研发投入逐年增高且失败风险逐年增大的趋势,挖掘已有药物新的适应症(旧药新用或药物重新定位)能够在一定程度上有效规避研发风险、降低研发成本、加快药物上市的步伐从而迅速满足临床用药需求,因而成为众多国际制药企业重视和采用的一种战略.随着药物生物信息学的发展,药物重定位策略正从依赖临床观察的经验性研究,朝着基于大规模组学和医药大数据分析的系统性研究转变.本文将着重介绍国际上一系列用于挖掘和指导药物重定位的医药大数据挖掘方法,如基于基因表达谱联系图、全基因组关联分析、化合物-蛋白互作组、药物副作用等.参考和借鉴上述各种前沿技术,将有助于我国科研工作者开发和改进适应我国国情的药物重定位技术,特别是中药的适应症拓展,从而为我国带来社会和经济效益.

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