Epigenetics Section, Mouse Cancer Genetics Program, Center for Cancer Research, National Cancer Institute, Frederick, MD 21702.
CCR Collaborative Bioinformatics Resource, Center for Cancer Research, National Cancer Institute, Bethesda, MD 20892.
Proc Natl Acad Sci U S A. 2020 Aug 18;117(33):20100-20108. doi: 10.1073/pnas.2004112117. Epub 2020 Jul 29.
Mutation of HELLS (Helicase, Lymphoid-Specific)/Lsh in human DNA causes a severe immunodeficiency syndrome, but the nature of the defect remains unknown. We assessed here the role of Lsh in hematopoiesis using conditional Lsh knockout mice with expression of Mx1 or Vav Cre-recombinase. Bone marrow transplantation studies revealed that Lsh depletion in hematopoietic stem cells severely reduced B cell numbers and impaired B cell development in a hematopoietic cell-autonomous manner. Lsh-deficient mice without bone marrow transplantation exhibited lower Ig levels in vivo compared to controls despite normal peripheral B cell numbers. Purified B lymphocytes proliferated normally but produced less immunoglobulins in response to in vitro stimulation, indicating a reduced capacity to undergo class switch recombination (CSR). Analysis of germline transcripts, examination of double-stranded breaks using biotin-labeling DNA break assay, and End-seq analysis indicated that the initiation of the recombination process was unscathed. In contrast, digestion-circularization PCR analysis and high-throughput sequencing analyses of CSR junctions and a chromosomal break repair assay indicated an impaired ability of the canonical end-joining pathway in Lsh-deficient B cells. Our data suggest a hematopoietic cell-intrinsic role of Lsh in B cell development and in CSR providing a potential target for immunodeficiency therapy.
HELLS(Helicase,Lymphoid-Specific)/Lsh 基因在人类 DNA 中的突变会导致严重的免疫缺陷综合征,但缺陷的性质仍不清楚。我们使用表达 Mx1 或 Vav Cre 重组酶的条件性 Lsh 敲除小鼠,评估了 Lsh 在造血中的作用。骨髓移植研究表明,造血干细胞中 Lsh 的耗竭以造血细胞自主的方式严重减少了 B 细胞数量并损害了 B 细胞发育。尽管外周 B 细胞数量正常,但未进行骨髓移植的 Lsh 缺陷小鼠体内的 Ig 水平低于对照。纯化的 B 淋巴细胞在体外刺激下正常增殖,但产生的免疫球蛋白较少,表明其类别转换重组(CSR)的能力降低。对生殖系转录本的分析、使用生物素标记 DNA 断裂测定法对双链断裂的检测,以及 End-seq 分析表明,重组过程的起始未受影响。相比之下,消化-环化 PCR 分析和 CSR 连接处的高通量测序分析以及染色体断裂修复测定表明,Lsh 缺陷 B 细胞中经典的末端连接途径的能力受损。我们的数据表明 Lsh 在 B 细胞发育和 CSR 中具有造血细胞内在的作用,为免疫缺陷治疗提供了一个潜在的靶点。