Institute of Immunology and Immunontherapy, Medical School, University of Birmingham, Edgbaston, B15 2TT, UK; UCB Pharma, Slough, Berkshire, SL1 3WE, UK.
Massachusetts General Hospital Cancer Center and Department of Medicine Harvard Medical School, 13th Street, Charlestown, MA, 02129, USA.
J Autoimmun. 2017 Jun;80:1-9. doi: 10.1016/j.jaut.2017.04.002. Epub 2017 Apr 8.
Immune dysregulation is a prominent feature of primary immunodeficiency disorders, which commonly manifested as autoimmunity, cytopenias and inflammatory bowel disease. In partial T-cell immunodeficiency disorders, it has been proposed that the imbalance between effector and regulatory T-cells drives the breakdown of peripheral tolerance. While there is no robust test for immune dysregulation, the T-cell receptor repertoire is used as a surrogate marker, and has been shown to be perturbed in a number of immunodeficiency disorders featuring immune dysregulation including Omenn's Syndrome, Wiskott-Aldrich Syndrome, and common variable immunodeficiency. This review discusses how recent advances in TCR next-generation sequencing and bioinformatics have led to the in-depth characterization of CDR3 sequences and an exponential growth in examinable parameters. Specifically, we highlight the use of junctional diversity as a means to differentiate intrinsic T-cell defects from secondary causes of repertoire perturbation in primary immunodeficiency disorders. However, key questions, such as the identity of antigenic targets for large, expanded T-cell clonotypes, remain unanswered despite the fact that such clones are likely to play a pathogenic role in driving immune dysregulation and autoimmunity. Finally, we discuss a number of emerging technologies such as in silico reconstruction, high-throughput pairwise αβ sequencing and single-cell RNAseq that offer the potential to define the antigenic epitope and function of a given T-cell, thereby enhancing our understanding in this field.
免疫失调是原发性免疫缺陷病的一个突出特征,通常表现为自身免疫、细胞减少症和炎症性肠病。在部分 T 细胞免疫缺陷病中,人们提出效应 T 细胞和调节性 T 细胞之间的失衡导致外周耐受的破坏。虽然目前还没有针对免疫失调的可靠检测方法,但 T 细胞受体库被用作替代标志物,并且已经在多种表现为免疫失调的免疫缺陷病中显示出紊乱,包括 Omenn 综合征、Wiskott-Aldrich 综合征和常见可变免疫缺陷。本文讨论了 TCR 下一代测序和生物信息学的最新进展如何导致 CDR3 序列的深入表征以及可检查参数的指数增长。具体来说,我们强调了使用连接多样性作为区分原发性免疫缺陷病中内在 T 细胞缺陷和反应库紊乱的继发原因的一种手段。然而,尽管这些克隆很可能在驱动免疫失调和自身免疫方面发挥致病作用,但仍存在一些关键问题尚未得到解答,例如针对大的、扩增的 T 细胞克隆型的抗原靶标的身份。最后,我们讨论了一些新兴技术,如计算重构、高通量成对的αβ测序和单细胞 RNAseq,它们有可能定义给定 T 细胞的抗原表位和功能,从而增强我们在这一领域的理解。