UC Berkeley-UC San Francisco Graduate Program in Bioengineering, and.
Department of Pharmaceutical Chemistry, UCSF, San Francisco, California, USA.
JCI Insight. 2024 May 14;9(12):e176963. doi: 10.1172/jci.insight.176963.
Juvenile dermatomyositis (JDM) is one of several childhood-onset autoimmune disorders characterized by a type I IFN response and autoantibodies. Treatment options are limited due to an incomplete understanding of how the disease emerges from dysregulated cell states across the immune system. We therefore investigated the blood of patients with JDM at different stages of disease activity using single-cell transcriptomics paired with surface protein expression. By immunophenotyping peripheral blood mononuclear cells, we observed skewing of the B cell compartment toward an immature naive state as a hallmark of JDM at diagnosis. Furthermore, we find that these changes in B cells are paralleled by T cell signatures suggestive of Th2-mediated inflammation that persist despite disease quiescence. We applied network analysis to reveal that hyperactivation of the type I IFN response in all immune populations is coordinated with previously masked cell states including dysfunctional protein processing in CD4+ T cells and regulation of cell death programming in NK cells, CD8+ T cells, and γδ T cells. Together, these findings unveil the coordinated immune dysregulation underpinning JDM and provide insight into strategies for restoring balance in immune function.
幼年特发性皮肌炎(JDM)是几种儿童期发病的自身免疫性疾病之一,其特征是存在 I 型干扰素反应和自身抗体。由于对疾病如何从免疫系统失调的细胞状态中出现的机制了解不完整,因此治疗选择有限。因此,我们使用单细胞转录组学与表面蛋白表达相结合,研究了处于不同疾病活动阶段的 JDM 患者的血液。通过免疫表型分析外周血单核细胞,我们观察到 B 细胞区室向幼稚原始状态倾斜,这是 JDM 诊断时的一个标志。此外,我们发现这些 B 细胞的变化与提示 Th2 介导炎症的 T 细胞特征平行存在,尽管疾病处于静止状态,但这些特征仍然存在。我们应用网络分析揭示了所有免疫群体中 I 型干扰素反应的过度激活与以前被掩盖的细胞状态相协调,包括 CD4+T 细胞中功能失调的蛋白处理以及 NK 细胞、CD8+T 细胞和γδ T 细胞中细胞死亡编程的调节。总之,这些发现揭示了 JDM 潜在的免疫失调的协调机制,并为恢复免疫功能平衡提供了策略。