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照亮可药物基因组:前进之路。

Illuminating the druggable genome: Pathways to progress.

作者信息

Sharma Karlie R, Colvis Christine M, Rodgers Griffin P, Sheeley Douglas M

机构信息

National Center for Advancing Translational Sciences, National Institutes of Health, 6701 Democracy Blvd, Bethesda, MD 20892, USA.

National Center for Advancing Translational Sciences, National Institutes of Health, 6701 Democracy Blvd, Bethesda, MD 20892, USA.

出版信息

Drug Discov Today. 2024 Mar;29(3):103805. doi: 10.1016/j.drudis.2023.103805. Epub 2023 Oct 27.

Abstract

There are ∼4500 genes within the 'druggable genome', the subset of the human genome that expresses proteins able to bind drug-like molecules, yet existing drugs only target a few hundred. A substantial subset of druggable proteins are largely uncharacterized or understudied, with many falling within G protein-coupled receptor (GPCR), ion channel, and kinase protein families. To improve scientific understanding of these three understudied protein families, the US National Institutes of Health launched the Illuminating the Druggable Genome Program. Now, as the program draws to a close, this review will lay out resources developed by the program that are intended to equip the scientific community with the tools necessary to explore previously understudied biology with the potential to rapidly impact human health.

摘要

在“可成药基因组”(即人类基因组中能够表达可与类药物分子结合的蛋白质的子集)中大约有4500个基因,但现有药物仅针对其中几百个基因。可成药蛋白质的很大一部分在很大程度上尚未得到表征或研究不足,其中许多属于G蛋白偶联受体(GPCR)、离子通道和激酶蛋白家族。为了增进对这三个研究不足的蛋白质家族的科学理解,美国国立卫生研究院启动了“照亮可成药基因组计划”。现在,随着该计划接近尾声,本综述将介绍该计划开发的资源,这些资源旨在为科学界提供必要的工具,以探索此前研究不足但有可能迅速影响人类健康的生物学领域。

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