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从现代药物研发的角度来看 ,发现并验证具有自主知识产权的药物靶标应该成为产生“重磅炸弹”式创新药物的源头 ,对于创新药物的研制可能具有决定性意义。所谓“药物靶标”是指存在于组织细胞内与药物相互作用 ,并赋予药物效应的特定分子。具体来说 ,作为药物靶标的蛋白质必须在病变细胞或组织中表达 ,并且在细胞培养体系中可以通过调节靶标活性改善相关表现 ,最后这些效应必须在疾病动物模型中再现。实际上 ,作为有价值的药物靶标必须获得有效的药物。人类基因组和蛋白质组学研究已经揭开了潜在药物靶标的面纱。但是 ,基因组和后基因组给人们带来大量信息的同时 ,也为药物靶标筛选带来了越来越大的困难。如何综合运用生物信息学、分子生物学、疾病发病学和药理学等方法发现和确证药物作用新靶标是目前面临的重要挑战。
From a modern drug discovery perspective, discovering and validating drug targets with proprietary IPRs should be the source of “blockbuster” innovative drugs that may have decisive implications for the development of innovative drugs. By “drug target” is meant a specific molecule present in a tissue cell that interacts with a drug and confers a drug effect. In particular, proteins that are targets of drugs must be expressed in diseased cells or tissues, and related manifestations can be improved by modulating the activity of the target in cell culture systems, eventually these effects must be reproduced in animal models of disease. In fact, effective medicines must be obtained as valuable drug targets. Human genome and proteomics research has uncovered the veil of potential drug targets. However, while genomics and post-genomics bring a lot of information to people, it also brings more and more difficulties in drug target screening. How to find out and confirm the new target of drug action through the combination of bioinformatics, molecular biology, disease epidemiology and pharmacology is an important challenge nowadays.