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在干细胞衍生的心肌细胞中可检测到不同药物模式导致的hERG阻断引起的延迟复极化:孵育时间很重要。

Delayed Repolarization Caused by hERG Block With Different Drug Modalities Can Be Detected in Stem Cell-Derived Cardiomyocytes: Incubation Time Matters.

作者信息

Qu Yusheng, Guo Wei, Wu Bin, Cui Jixin, Gao BaoXi, Nichols Jill V, Vargas Hugo M

机构信息

Translational Safety & Bioanalytical Sciences, Amgen Research, Thousand Oaks, California, USA.

Cardiometabolic Disorders, Amgen Research, Thousand Oaks, California, USA.

出版信息

Clin Transl Sci. 2025 Sep;18(9):e70283. doi: 10.1111/cts.70283.

DOI:10.1111/cts.70283
PMID:40848285
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12374734/
Abstract

The intrinsic characteristics of oligonucleotides pose a challenge for their assessment in conventional primary in vitro cardiac models, which were designed for the acute application of small molecule agents and are not suitable for transfection and extended culture periods. Conversely, human-induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CM) offer a viable platform for the evaluation of agents over prolonged application and recording times. Our previous experiments demonstrated that a chronic protocol of 48 h is necessary to discern the functional effects of a siRNA targeting hERG in a stable cell line heterologously expressing hERG. To investigate whether a targeted hERG siRNA induces delayed repolarization in hiPSC-CM, we recorded field potentials (FPs) using a multielectrode array. FP duration (FPD) prolongation was noted as early as 10 min after exposure to moxifloxacin, whereas pentamidine required 24 h to induce FPD prolongation. Transfection with hERG-targeting siRNA reduced mRNA expression at 6 h post-transfection. However, FPD prolongation was only observed after 24 h post-transfection, with significantly larger effects at 48 h, which is indicative of the time needed for turnover of the hERG protein on the plasma membrane. Our findings provide compelling evidence that MEA recordings in hiPSC-CM can accurately detect disruptions in cardiac repolarization due to various mechanisms that impair hERG channel function, including direct channel blockade, inhibition of protein trafficking, and gene silencing via siRNA. The findings also indicate that indirect mechanisms of hERG knockdown, including gene silencing, require assessment at least 48 h following treatment to detect delayed repolarization in the hiPSC-CM model.

摘要

寡核苷酸的内在特性对其在传统的体外心脏原代模型中的评估构成了挑战,这些模型是为小分子药物的急性应用而设计的,不适用于转染和延长培养时间。相反,人诱导多能干细胞衍生的心肌细胞(hiPSC-CM)为长时间应用和记录药物提供了一个可行的平台。我们之前的实验表明,对于在稳定异源表达hERG的细胞系中识别靶向hERG的siRNA的功能效应,48小时的慢性方案是必要的。为了研究靶向hERG的siRNA是否会在hiPSC-CM中诱导延迟复极化,我们使用多电极阵列记录了场电位(FP)。早在暴露于莫西沙星后10分钟就观察到FP持续时间(FPD)延长,而喷他脒则需要24小时才能诱导FPD延长。转染靶向hERG的siRNA在转染后6小时降低了mRNA表达。然而,FPD延长仅在转染后24小时观察到,在48小时时效应显著更大,这表明质膜上hERG蛋白周转所需的时间。我们的研究结果提供了令人信服的证据,即hiPSC-CM中的MEA记录可以准确检测由于各种损害hERG通道功能的机制(包括直接通道阻断、蛋白质转运抑制和通过siRNA的基因沉默)导致的心脏复极化中断。研究结果还表明,hERG敲低的间接机制,包括基因沉默,需要在治疗后至少48小时进行评估,以检测hiPSC-CM模型中的延迟复极化。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8252/12374734/c3161716e818/CTS-18-e70283-g006.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8252/12374734/c3161716e818/CTS-18-e70283-g006.jpg

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本文引用的文献

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