Department of Physiology and Pharmacology, University of Western Ontario, London, Ontario, Canada N6A 5C1.
Division of BioMedical Sciences, Faculty of Medicine, Memorial University of Newfoundland, St. John's, Newfoundland and Labrador, Canada A1B 3V6.
Biochem J. 2024 Jun 19;481(12):741-758. doi: 10.1042/BCJ20240016.
Mutations in more than half of human connexin genes encoding gap junction (GJ) subunits have been linked to inherited human diseases. Functional studies of human GJ channels are essential for revealing mechanistic insights into the etiology of disease-linked connexin mutants. However, the commonly used Xenopus oocytes, N2A, HeLa, and other model cells for recombinant expression of human connexins have different and significant limitations. Here we developed a human cell line (HEK293) with each of the endogenous connexins (Cx43 and Cx45) knocked out using the CRISPR-Cas9 system. Double knockout HEK293 cells showed no background GJ coupling, were easily transfected with several human connexin genes (such as those encoding Cx46, Cx50, Cx37, Cx45, Cx26, and Cx36) which successfully formed functional GJs and were readily accessible for dual patch clamp analysis. Single knockout Cx43 or Cx45 HEK cell lines could also be used to characterize human GJ channels formed by Cx45 or Cx43, respectively, with an expression level suitable for studying macroscopic and single channel GJ channel properties. A cardiac arrhythmia linked Cx45 mutant R184G failed to form functional GJs in DKO HEK293 cells with impaired localizations. These genetically engineered HEK293 cells are well suited for patch clamp study of human GJ channels.
超过一半的人类连接蛋白基因(encoding gap junction (GJ) subunits)的突变与遗传性人类疾病有关。研究人类 GJ 通道的功能对于揭示与连接蛋白突变相关疾病的发病机制至关重要。然而,常用于重组表达人类连接蛋白的非洲爪蟾卵母细胞、N2A、HeLa 和其他模式细胞存在不同且显著的局限性。在这里,我们使用 CRISPR-Cas9 系统敲除了内源性连接蛋白(Cx43 和 Cx45),开发了一种人细胞系(HEK293)。双敲除 HEK293 细胞没有背景 GJ 偶联,很容易转染几种人类连接蛋白基因(如编码 Cx46、Cx50、Cx37、Cx45、Cx26 和 Cx36 的基因),这些基因成功地形成了功能性 GJ,并且很容易进行双膜片钳分析。单敲除 Cx43 或 Cx45 的 HEK 细胞系也可用于分别表征由 Cx45 或 Cx43 形成的人类 GJ 通道,其表达水平适合研究宏观和单通道 GJ 通道特性。一种与心律失常相关的 Cx45 突变体 R184G 无法在 DKO HEK293 细胞中形成功能性 GJ,其定位受损。这些基因工程 HEK293 细胞非常适合用于人类 GJ 通道的膜片钳研究。