Romero Lucia, Trenor Beatriz, Yang Pei-Chi, Saiz Javier, Clancy Colleen E
J Mol Cell Cardiol. 2015 Oct;87:271-82. doi: 10.1016/j.yjmcc.2015.08.015.
Accurate diagnosis of predisposition to long QT syndrome is crucial for reducing the risk of cardiac arrhythmias. In recent years, drug-induced provocative tests have proved useful to unmask some latent mutations linked to cardiac arrhythmias. In this study we expanded this concept by developing a prototype for a computational provocative screening test to reveal genetic predisposition to acquired long-QT syndrome (aLQTS). We developed a computational approach to reveal the pharmacological properties of I(Kr) blocking drugs that are most likely to cause aLQTS in the setting of subtle alterations in I(Kr) channel gating that would be expected to result from benign genetic variants.Weused themodel to predict themost potentially lethal combinations of kinetic anomalies and drug properties. In doing so, we also implicitly predicted ideal inverse therapeutic properties of K channel openers that would be expected to remedy a specific defect. We systematically performed “in silico mutagenesis” by altering discrete kinetic transition rates of the Fink et al. Markov model of human I(Kr) channels, corresponding to activation, inactivation, deactivation and recovery from inactivation of I(Kr) channels. We then screened and identified the properties of I(Kr) blockers that caused acquired long QT and therefore unmasked mutant phenotypes formild,moderate and severe variants. Mutant I(Kr) channels were incorporated into the O'Hara et al. human ventricular action potential (AP) model and subjected to simulated application of a wide variety of I(Kr)-drug interactions in order to identify the characteristics that selectively exacerbate the AP duration (APD) differences between wild-type and I(Kr) mutated cells. Our results show that drugs with disparate affinities to conformation states of the I(Kr) channel are key to amplify variants underlying susceptibility to acquired long QT syndrome, an effect that is especially pronounced at slow frequencies. Finally, we developed a mathematical formulation of the M54T MiRP1 latent mutation and simulated a provocative test. In this setting, application of dofetilide dramatically amplified the predicted QT interval duration in the M54T hMiRP1 mutation compared to wild-type.
准确诊断长QT综合征的易感性对于降低心律失常风险至关重要。近年来,药物激发试验已被证明有助于揭示一些与心律失常相关的潜在突变。在本研究中,我们通过开发一种计算激发筛选试验的原型来扩展这一概念,以揭示获得性长QT综合征(aLQTS)的遗传易感性。我们开发了一种计算方法,以揭示I(Kr)阻断药物的药理学特性,这些药物在I(Kr)通道门控发生细微改变(预计由良性基因变异引起)的情况下最有可能导致aLQTS。我们使用该模型预测动力学异常和药物特性的最具潜在致死性的组合。在此过程中,我们还隐含地预测了钾通道开放剂的理想反向治疗特性,预计这些特性可纠正特定缺陷。我们通过改变人类I(Kr)通道的Fink等人马尔可夫模型的离散动力学转换速率,系统地进行了“计算机模拟诱变”,这些转换速率对应于I(Kr)通道的激活、失活、去激活和从失活状态恢复。然后,我们筛选并确定了导致获得性长QT的I(Kr)阻断剂的特性,从而揭示了轻度、中度和重度变异的突变表型。将突变的I(Kr)通道纳入O'Hara等人的人类心室动作电位(AP)模型,并对各种I(Kr)-药物相互作用进行模拟应用,以确定选择性加剧野生型和I(Kr)突变细胞之间动作电位时程(APD)差异的特征。我们的结果表明,对I(Kr)通道构象状态具有不同亲和力的药物是放大获得性长QT综合征易感性潜在变异的关键,这种效应在低频时尤为明显。最后,我们开发了M54T MiRP1潜在突变的数学公式,并模拟了激发试验。在这种情况下,与野生型相比,多非利特的应用显著放大了M54T hMiRP1突变中预测的QT间期持续时间。