药物转运体作为调控药物跨膜转运的核心膜蛋白，通过ATP水解或离子/浓度梯度驱动的运输机制，介导药物、内源性生物活性分子及外源性毒素的跨膜转运过程，在药物吸收、分布、代谢与排泄过程中发挥关键作用。这类蛋白主要分为ATP结合盒转运体和溶质载体转运体两大家族。研究表明，转运体基因的单核苷酸多态性可通过改变其表达水平、转运活性或底物亲和力，显著影响药物的动力学特征及疗效与安全性。近年来，随着药物基因组学的快速发展，相关研究已从分子机制解析逐步延伸至临床实践。系统综述与药物处置密切相关的转运体基因多态性，重点解析其对药动学参数、临床疗效及不良反应的影响，旨在为精准用药提供遗传学依据，推动个体化治疗策略的优化。

Drug transporters, as core membrane proteins regulating the transmembrane transport of drugs, mediate the transmembrane transport of drugs, endogenous bioactive molecules, and exogenous toxins through ATP hydrolysis or ion/concentration gradient-driven transport mechanisms, playing a crucial role in drug absorption, distribution, metabolism, and excretion. These proteins are mainly classified into two families: ATP-binding cassette transporters and solute carrier transporters. Studies have shown that single nucleotide polymorphisms in transporter genes can significantly affect the pharmacokinetic characteristics and efficacy and safety of drugs by altering their expression levels, transport activities, or substrate affinities. In recent years, with the rapid development of pharmacogenomics, related research has gradually extended from molecular mechanism analysis to clinical practice. This systematic review focuses on the polymorphisms of transporters closely related to drug disposition, particularly their impact on pharmacokinetic parameters, clinical efficacy, and adverse reactions, aiming to provide genetic evidence for precision medicine and promote the optimization of personalized therapeutic strategies.

R969.3

药物成药性评价与系统转化全国重点实验室(天津药物研究院)自主研究课题资助项目(712024001)