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机构地区:[1]军事医学科学院生物工程研究所,北京100071
出 处:《药学学报》2009年第7期687-694,共8页Acta Pharmaceutica Sinica
摘 要:模式生物秀丽隐杆线虫(Caenorhabditis elegans)对基础生物学研究贡献卓著。随着功能基因组时代的到来,线虫被视为可借以探明人类致病基因或致病相关因子,寻找可能的药物靶点的强大工具。这得益于线虫的丰富的可利用生物学信息,灵活多样的遗传学可操作性,简易的培养维持条件,尤其是其独特的生理特点适合于大规模化学药物的体内筛选或全基因组功能研究。本文将从新药研发的角度总结线虫的特点,与人类基因的同源性,讨论以线虫为技术平台的药物研发主要方法,以及成功案例,并探讨线虫在医药学领域的发展热点。A simple model organism Caenorhabditis elegans has contributed substantially to the fundamental researches in biology. In an era of functional genomics, nematode worm has been developed into a multi-purpose tool that can be exploited to identify disease-causing or disease-associated genes, validate potential drug targets. This, coupled with its genetic amenability, low cost experimental manipulation and compatibility with high throughput screening in an intact physiological condition, makes the model organism into an effective toolbox for drug discovery. This review shows the unique features of C. elegans, how it can play a valuable role in our understanding of the molecular mechanism of human diseases and finding drug leads in drug development process.
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