Buchanan Ocular Therapeutics Unit, Department of Ophthalmology, New Zealand National Eye Centre, University of Auckland, Auckland, New Zealand.
Department of Ophthalmology, New Zealand National Eye Centre, University of Auckland, Auckland, New Zealand.
Drug Deliv Transl Res. 2020 Jun;10(3):751-765. doi: 10.1007/s13346-020-00763-y.
Hypoxic injury results in cell death, tissue damage and activation of inflammatory pathways. This is mediated by pathological Connexin43 (Cx43) hemichannel (HC) opening resulting in osmotic and ionic imbalances as well as cytokine production perpetuating the inflammatory environment. Gap19 is an intracellularly acting Cx43 mimetic peptide that blocks HC opening and thus promotes cell survival. However, native Gap19, which must enter the cell in order to function, exhibits low cell permeability. In this study, Gap19 was conjugated to the cell-penetrating peptide, Xentry, to investigate if cellular uptake could be improved while maintaining peptide function. Cellular uptake of Xentry-Gap19 (XG19) was much greater than that of native Gap19 even under normal cell culture conditions. Peptide function was maintained post uptake as shown by reduced ethidium homodimer influx and ATP release due to Cx43 HC block. While XG19 blocked pathologic HC opening though, normal gap junction communication required for cell repair and survival mechanisms was not affected as shown in a dye scrape-load assay. Under hypoxic conditions, increased expression of Syndecan-4, a plasma membrane proteoglycan targeted by Xentry, enabled even greater XG19 uptake leading to higher inhibition of ATP release and greater cell survival. This suggests that XG19, which is targeted specifically to hypoxic cells, can efficiently and safely block Cx43 HC and could therefore be a novel treatment for hypoxic and inflammatory diseases. Graphical abstract.
缺氧损伤导致细胞死亡、组织损伤和炎症途径的激活。这是由病理连接蛋白 43 (Cx43) 半通道 (HC) 开放介导的,导致渗透和离子失衡以及细胞因子产生,从而维持炎症环境。Gap19 是一种细胞内作用的 Cx43 模拟肽,可阻断 HC 开放,从而促进细胞存活。然而,必须进入细胞才能发挥作用的天然 Gap19,其细胞通透性较低。在这项研究中,Gap19 与穿膜肽 Xentry 缀合,以研究在保持肽功能的同时是否可以提高细胞摄取率。即使在正常的细胞培养条件下,Xentry-Gap19 (XG19) 的细胞摄取率也远高于天然 Gap19。摄取后肽功能得以维持,这表现为由于 Cx43 HC 阻断,细胞内 ethidium homodimer 流入和 ATP 释放减少。虽然 XG19 阻断了病理 HC 开放,但对细胞修复和存活机制所需的正常间隙连接通讯没有影响,如染料划痕加载测定所示。在缺氧条件下,连接蛋白聚糖-4 的表达增加,这是 Xentry 的靶标,从而使 XG19 的摄取率更高,导致 ATP 释放抑制率更高和细胞存活率更高。这表明,专门针对缺氧细胞的 XG19 可以有效地和安全地阻断 Cx43 HC,因此可能成为缺氧和炎症性疾病的一种新的治疗方法。