Department of Cardiology, Shanghai Chest Hospital, Shanghai Jiao Tong University, Shanghai 200030, China.
Department of Cardiology, Shanghai Fifth People's Hospital, Fudan University, Shanghai 200240, China.
Genes (Basel). 2021 Mar 12;12(3):408. doi: 10.3390/genes12030408.
Atrial fibrillation (AF) represents the most common type of clinical cardiac arrhythmia and substantially increases the risks of cerebral stroke, heart failure and death. Accumulating evidence has convincingly demonstrated the strong genetic basis of AF, and an increasing number of pathogenic variations in over 50 genes have been causally linked to AF. Nevertheless, AF is of pronounced genetic heterogeneity, and the genetic determinants underpinning AF in most patients remain obscure. In the current investigation, a Chinese pedigree with AF as well as ventricular arrhythmias and hypertrophic cardiomyopathy was recruited. Whole exome sequencing and bioinformatic analysis of the available family members were conducted, and a novel heterozygous variation in the KLF15 gene (encoding Krüppel-like factor 15, a transcription factor critical for cardiac electrophysiology and structural remodeling), NM_014079.4: c.685A>T; p.(Lys229*), was identified. The variation was verified by Sanger sequencing and segregated with autosomal dominant AF in the family with complete penetrance. The variation was absent from 300 unrelated healthy subjects used as controls. In functional assays using a dual-luciferase assay system, mutant KLF15 showed neither transcriptional activation of the KChIP2 promoter nor transcriptional inhibition of the CTGF promoter, alone or in the presence of TGFB1, a key player in the pathogenesis of arrhythmias and cardiomyopathies. The findings indicate KLF15 as a new causative gene responsible for AF as well as ventricular arrhythmias and hypertrophic cardiomyopathy, and they provide novel insight into the molecular mechanisms underlying cardiac arrhythmias and hypertrophic cardiomyopathy.
心房颤动(AF)是最常见的临床心律失常类型,显著增加了脑卒、心力衰竭和死亡的风险。越来越多的证据令人信服地证明了 AF 的强烈遗传基础,超过 50 个基因中的许多致病性变异与 AF 有因果关系。然而,AF 具有明显的遗传异质性,大多数患者中 AF 的遗传决定因素仍不清楚。在目前的研究中,我们招募了一个具有 AF 以及室性心律失常和肥厚型心肌病的中国家系。对现有的家族成员进行了全外显子组测序和生物信息学分析,并在 KLF15 基因(编码心脏电生理和结构重塑关键的转录因子 Krüppel-like factor 15)中发现了一个新的杂合变异,NM_014079.4:c.685A>T;p.(Lys229*)。该变异通过 Sanger 测序得到验证,并在家系中与常染色体显性遗传的 AF 完全共分离。该变异在 300 名作为对照的无关健康个体中不存在。在使用双荧光素酶检测系统的功能测定中,突变型 KLF15 既没有激活 KChIP2 启动子的转录激活作用,也没有抑制 CTGF 启动子的转录抑制作用,无论是单独作用还是在心律失常和心肌病发病机制中的关键因子 TGFB1 存在的情况下。这些发现表明 KLF15 是导致 AF 以及室性心律失常和肥厚型心肌病的新的致病基因,并为心律失常和肥厚型心肌病的分子机制提供了新的见解。