无义突变通读药物研发进展

发布时间：2018-04-11 05:23

本文选题：无义突变 + 提前终止密码子　；参考：《中国新药杂志》2017年08期

【摘要】：无义突变与众多疾病发生有关,包括肿瘤和遗传病。某些化合物如氨基糖苷类抗生素、非氨基糖苷类抗生素及其他小分子化合物能诱导无义突变通读,从而翻译出有功能的全长蛋白。化合物诱导的无义突变通读对于遗传病及肿瘤治疗是一个非常有潜力的治疗策略,但目前没有几种化合物显示出确切的临床疗效。因此,急需筛选新型、更具药物特性的化合物,推动通读治疗走向临床。本文详细介绍了无义突变通读药物的研究现状,为新药开发提供参考。
[Abstract]:Nonsense mutations are associated with many diseases, including neoplasms and genetic diseases.Some compounds, such as aminoglycoside antibiotics, non-aminoglycoside antibiotics and other small molecular compounds, can induce meaningless mutations to read through and translate functional full-length proteins.Compound induced nonsense mutation reading is a potential therapeutic strategy for hereditary diseases and tumor therapy, but few compounds have shown definite clinical efficacy.Therefore, the screening of new, more drug-specific compounds is urgently needed to promote read-through therapy to clinical use.In this paper, the current situation of research on nonsense mutants is introduced in detail, which provides a reference for the development of new drugs.
【作者单位】：武汉科技大学附属天佑医院中心实验室;武汉科技大学医学院公共卫生学院;
【基金】：国家自然科学基金资助项目(81373302) 武汉中青年医学骨干人才培养工程
【分类号】：R91

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