Department of Pediatric Hematology/Oncology, IRCCS, Ospedale Pediatrico Bambino Gesù, Viale San Paolo 15, 00146 Rome, Italy.
Rheumatology and Clinical Immunology, Humanitas Clinical and Research Center-IRCCS - Via Alessandro Manzoni, 56, 20089 Rozzano, Milan, Italy.
Int J Mol Sci. 2019 Sep 4;20(18):4337. doi: 10.3390/ijms20184337.
Systemic sclerosis (SSc) is a rare autoimmune disease, characterized by vasculopathy and fibrosis of the skin and internal organs. This disease is still considered incurable and is associated with a high risk of mortality, which is related to fibrotic events. An early diagnosis is useful for preventing complications, and targeted therapies reduce disease progression and ameliorate patients' quality of life. Nevertheless, there are no validated biomarkers for early diagnosis with predictive prognostic value. Exosomes are membrane vesicles, transporting proteins and nucleic acids that may be delivered to target cells, which influences cellular behavior. They play important roles in cell-cell communication, both in physiological and pathological conditions, and may be useful as circulating biomarkers. Recent evidences suggest a role for these microvesicles in the three main aspects related to the pathogenesis of SSc (immunity, vascular damage, and fibrosis). Moreover, exosomes are of particular interest in the field of nano-delivery and are used as biological carriers. In this review, we report the latest information concerning SSc pathogenesis, clinical aspects of SSc, and current approaches to the treatment of SSc. Furthermore, we indicate a possible role of exosomes in SSc pathogenesis and suggest their potential use as diagnostic and prognostic biomarkers, as well as therapeutic tools.
系统性硬化症(SSc)是一种罕见的自身免疫性疾病,其特征为皮肤和内脏器官的血管病变和纤维化。这种疾病仍然被认为是无法治愈的,并且与高死亡率相关,这与纤维化事件有关。早期诊断有助于预防并发症,而靶向治疗可减缓疾病进展并改善患者的生活质量。然而,目前尚无具有预测预后价值的早期诊断的验证性生物标志物。外泌体是一种膜囊泡,可运输蛋白质和核酸,并可传递给靶细胞,从而影响细胞行为。它们在细胞间通讯中发挥着重要作用,无论是在生理还是病理条件下,并且可能作为循环生物标志物有用。最近的证据表明,这些微囊泡在 SSc 的三个主要发病机制相关方面(免疫、血管损伤和纤维化)中起作用。此外,外泌体在纳米递药领域具有特殊的意义,并且被用作生物载体。在这篇综述中,我们报告了与 SSc 发病机制、SSc 的临床方面以及 SSc 的治疗方法有关的最新信息。此外,我们还指出了外泌体在 SSc 发病机制中的可能作用,并提出了将其用作诊断和预后生物标志物以及治疗工具的可能性。