CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.
Blood. 2013 Apr 18;121(16):3112-6. doi: 10.1182/blood-2012-10-460741. Epub 2013 Jan 14.
Primary B-cell disorders comprise a heterogeneous group of inherited immunodeficiencies, often associated with autoimmunity causing significant morbidity. The underlying genetic etiology remains elusive in the majority of patients. In this study, we investigated a patient from a consanguineous family suffering from recurrent infections and severe lupuslike autoimmunity. Immunophenotyping revealed progressive decrease of CD19(+) B cells, a defective class switch indicated by low numbers of IgM- and IgG-memory B cells, as well as increased numbers of CD21(low) B cells. Combined homozygosity mapping and exome sequencing identified a biallelic splice-site mutation in protein C kinase δ (PRKCD), causing the absence of the corresponding protein product. Consequently, phosphorylation of myristoylated alanine-rich C kinase substrate was decreased, and mRNA levels of nuclear factor interleukin (IL)-6 and IL-6 were increased. Our study uncovers human PRKCD deficiency as a novel cause of common variable immunodeficiency-like B-cell deficiency with severe autoimmunity.
原发性 B 细胞疾病包括一组异质性的遗传性免疫缺陷,常伴有导致严重发病率的自身免疫。在大多数患者中,潜在的遗传病因仍难以捉摸。在这项研究中,我们研究了一名来自近亲家庭的患者,该患者反复感染并患有严重的狼疮样自身免疫。免疫表型显示 CD19(+)B 细胞逐渐减少,IgM-和 IgG-记忆 B 细胞数量减少表明类别转换缺陷,以及 CD21(低)B 细胞数量增加。联合纯合子作图和外显子组测序鉴定出蛋白 C 激酶 δ (PRKCD)的双等位基因剪接位点突变,导致相应的蛋白产物缺失。因此,肉豆蔻酰化丙氨酸丰富的 C 激酶底物的磷酸化减少,核因子白细胞介素 (IL)-6 和 IL-6 的 mRNA 水平增加。我们的研究揭示了人类 PRKCD 缺陷是一种新型的常染色体显性免疫缺陷样 B 细胞缺陷伴严重自身免疫的原因。