Nagib Lydia, Kumar Anshul Sheel, Stratton Richard
Centre for Rheumatology and Connective Tissue Diseases, UCL Division of Medicine, London NW32PF, UK.
Cells. 2025 Jun 24;14(13):962. doi: 10.3390/cells14130962.
Systemic sclerosis (SSc) is an autoimmune fibrotic disorder affecting the skin and internal organs, categorized as either limited cutaneous SSc, where distal areas of skin are involved, or diffuse cutaneous SSc, where more extensive proximal skin involvement is seen. Vascular remodelling and internal organ involvement are frequent complications in both subsets. Multiple pathogenic mechanisms have been demonstrated, including production of disease-specific autoantibodies, endothelial cell damage at an early stage, infiltration of involved tissues by immune cells, as well as environmental factors triggering the onset such as solvents and viruses. Although not strongly familial, susceptibility to SSc is associated with multiple single nucleotide polymorphisms in immunoregulatory genes relevant to antigen presentation, T cell signalling and adaptive immunity, as well as innate immunity. In addition, several lines of evidence demonstrate abnormalities within the epithelial cell layer in SSc. Macroscopically, the SSc epidermis is pigmented, thickened and stiff and strongly promotes myofibroblasts in co-culture. Moreover, multiple activating factors and pathways have been implicated in the disease epidermis, including wound healing responses, induction of damage associated molecular patterns (DAMPS) and the release of pro-fibrotic growth factors and cytokines. Similar to SSc, data from studies of cutaneous wound healing indicate a major role for epidermal keratinocytes in regulating local fibroblast responses during repair of the wound defect. Since the epithelium is strongly exposed to environmental factors and richly populated with protective immune cells, it is possible that disease-initiating mechanisms in SSc involve dysregulated immunity and tissue repair within this cell layer. Treatments designed to restore epithelial homeostasis or else disrupt epithelial-fibroblast cross-talk could be of benefit in this severe and resistant disease. Accordingly, single cell analysis has confirmed an active signature in SSc keratinocytes, which was partially reversed following a period of JAK inhibitor therapy.
系统性硬化症(SSc)是一种影响皮肤和内脏器官的自身免疫性纤维化疾病,可分为局限性皮肤型SSc(累及皮肤远端区域)或弥漫性皮肤型SSc(近端皮肤受累范围更广)。血管重塑和内脏器官受累是这两种亚型常见的并发症。已证实多种致病机制,包括产生疾病特异性自身抗体、早期内皮细胞损伤、免疫细胞浸润受累组织,以及触发疾病发作的环境因素,如溶剂和病毒。虽然SSc并非强家族性疾病,但其易感性与免疫调节基因中的多个单核苷酸多态性相关,这些基因与抗原呈递、T细胞信号传导和适应性免疫以及固有免疫有关。此外,多条证据表明SSc上皮细胞层存在异常。宏观上,SSc表皮色素沉着、增厚且僵硬,在共培养中强烈促进肌成纤维细胞生成。此外,多种激活因子和信号通路与疾病表皮有关,包括伤口愈合反应、损伤相关分子模式(DAMPS)的诱导以及促纤维化生长因子和细胞因子的释放。与SSc类似,皮肤伤口愈合研究数据表明,表皮角质形成细胞在伤口缺损修复过程中调节局部成纤维细胞反应方面起主要作用。由于上皮细胞强烈暴露于环境因素且富含保护性免疫细胞,SSc的疾病起始机制可能涉及该细胞层内免疫失调和组织修复。旨在恢复上皮细胞稳态或破坏上皮-成纤维细胞相互作用的治疗方法可能对这种严重且难治的疾病有益。因此,单细胞分析证实了SSc角质形成细胞中的活跃特征,在接受一段时间的JAK抑制剂治疗后部分得到逆转。