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靶向 microRNA 用于药物研发:一种新范式。

Targeting miRNAs for drug discovery: a new paradigm.

机构信息

Department of Biotechnology and Bioinformatics, New Drug Discovery Research (NDDR), Ranbaxy Laboratories Limited, Gurgaon, Haryana, India.

出版信息

Curr Mol Med. 2010 Jul;10(5):503-10. doi: 10.2174/156652410791608216.

Abstract

The discovery of miRNAs and the establishment of it's clinical links with multiple diseases has led to a paradigm shift in the drug development pipeline of major pharmaceutical companies and has given birth to several biotechnology enterprises revolving around these magic molecules. The miRNA profiling studies over the last few years have indicated implicit involvement of miRNAs in the pathobiology of cancer, diabetes, infectious diseases as well as cardiovascular, neurological and immune system disorders. This information is currently being translated into tools for diagnosis, prognosis and predicting response to treatment. In addition, active and vigorous investigations are ongoing in several laboratories across academia and industry to decipher the precise molecular functions and mechanism of action for key miRNAs with therapeutic potential. Knowledge gained from these efforts will surely pave the way for designing effective R&D strategies and will revolutionize the way we diagnose and treat various diseases. The present review attempts to track the evolutionary progression of microRNA research from it's early infancy years to its maturity into a dynamic field of drug discovery.

摘要

miRNAs 的发现及其与多种疾病的临床联系的确立,导致了大型制药公司药物研发管道的范式转变,并催生了几家围绕这些神奇分子的生物技术企业。过去几年的 miRNA 分析研究表明,miRNAs 在内分泌、心血管、神经和免疫系统疾病以及癌症、糖尿病和传染病的病理生物学中具有潜在的作用。这些信息目前正在转化为用于诊断、预后和预测治疗反应的工具。此外,学术界和工业界的几个实验室正在积极开展研究,以揭示具有治疗潜力的关键 miRNA 的精确分子功能和作用机制。这些努力所获得的知识必将为设计有效的研发策略铺平道路,并将彻底改变我们诊断和治疗各种疾病的方式。本综述试图追踪 miRNA 研究从早期婴儿期到成熟为药物发现领域的动态领域的发展历程。

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