Kralj Thomas, Brouwer Kim L R, Creek Darren J
Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences, Monash University, Parkville, VIC, Australia.
Division of Pharmacotherapy and Experimental Therapeutics, UNC Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599-7569, USA.
Toxicol Sci. 2021 Aug 30;183(1):1-13. doi: 10.1093/toxsci/kfab069.
Drug-induced liver injury (DILI) is a significant clinical issue, affecting 1-1.5 million patients annually, and remains a major challenge during drug development-toxicity and safety concerns are the second-highest reason for drug candidate failure. The future prevalence of DILI can be minimized by developing a greater understanding of the biological mechanisms behind DILI. Both qualitative and quantitative analytical techniques are vital to characterizing and investigating DILI. In vitro assays are capable of characterizing specific aspects of a drug's hepatotoxic nature and multiplexed assays are capable of characterizing and scoring a drug's association with DILI. However, an even deeper insight into the perturbations to biological pathways involved in the mechanisms of DILI can be gained through the use of omics-based analytical techniques: genomics, transcriptomics, proteomics, and metabolomics. These omics analytical techniques can offer qualitative and quantitative insight into genetic susceptibilities to DILI, the impact of drug treatment on gene expression, and the effect on protein and metabolite abundance. This review will discuss the analytical techniques that can be applied to characterize and investigate the biological mechanisms of DILI and potential predictive biomarkers.
药物性肝损伤(DILI)是一个重大的临床问题,每年影响100万至150万患者,并且在药物研发过程中仍然是一个主要挑战——毒性和安全性问题是候选药物失败的第二大原因。通过更深入地了解DILI背后的生物学机制,可以将DILI未来的患病率降至最低。定性和定量分析技术对于表征和研究DILI至关重要。体外试验能够表征药物肝毒性性质的特定方面,而多重试验能够表征药物与DILI的关联并进行评分。然而,通过使用基于组学的分析技术:基因组学、转录组学、蛋白质组学和代谢组学,可以更深入地了解DILI机制中涉及的生物途径的扰动。这些组学分析技术可以提供关于DILI遗传易感性、药物治疗对基因表达的影响以及对蛋白质和代谢物丰度的影响的定性和定量见解。本综述将讨论可用于表征和研究DILI生物学机制及潜在预测生物标志物的分析技术。