Immunology, Allergy and Rheumatology Section, Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, Tex.
Division of Immunology, Boston Children's Hospital, and the Departments of Pediatrics and Pathology, Harvard Medical School, Boston, Mass.
J Allergy Clin Immunol. 2015 May;135(5):1132-41. doi: 10.1016/j.jaci.2015.02.037.
Genetic identification of immunodeficiency syndromes has become more efficient with the availability of whole-exome sequencing, expediting the identification of relevant genes and complementing traditional linkage analysis and homozygosity mapping. New genes defects causing immunodeficiency include phophoglucomutase 3 (PGM3), cytidine 5' triphosphate synthase 1 (CTPS1), nuclear factor κB-inducing kinase (NIK), cytotoxic T lymphocyte-associated antigen 4 (CTLA4), B-cell chronic lymphocytic leukemia/lymphoma 10 (BCL10), phosphoinositide-3 kinase regulatory subunit 1 (PIK3R1), IL21, and Jagunal homolog 1 (JAGN1). New case reports expanded the clinical spectrum of gene defects. For example, a specific recombination-activating gene 1 variant protein with partial recombinant activity might produce Omenn syndrome or a common variable immunodeficiency phenotype. Central and peripheral B-cell tolerance was investigated in patients with several primary immunodeficiencies, including common variable immunodeficiency and Wiskott-Aldrich syndrome, to explain the occurrence of autoimmunity and inflammatory disorders. The role of IL-12 and IL-15 in the enhancement of natural killer cell activity was reported. Newborn screening for T-cell deficiency is being implemented in more states and is achieving its goal of defining the true incidence of severe combined immunodeficiency and providing early treatment that offers the highest survival for these patients. Definitive treatment of severe immunodeficiency with both hematopoietic stem cell transplantation and gene therapy was reported to be successful, with increasing definition of conditions needed for optimal outcomes. Progress in HIV infection is directed toward the development of an effective vaccine and the eradication of hidden latent virus reservoirs.
随着外显子组测序的应用,免疫缺陷综合征的基因鉴定变得更加高效,加速了相关基因的鉴定,并补充了传统的连锁分析和纯合性作图。导致免疫缺陷的新基因缺陷包括磷酸葡萄糖变位酶 3(PGM3)、胞苷 5'-三磷酸合酶 1(CTPS1)、核因子κB 诱导激酶(NIK)、细胞毒性 T 淋巴细胞相关抗原 4(CTLA4)、B 细胞慢性淋巴细胞白血病/淋巴瘤 10(BCL10)、磷酸肌醇 3 激酶调节亚基 1(PIK3R1)、IL21 和 Jagunal 同源物 1(JAGN1)。新的病例报告扩展了基因缺陷的临床谱。例如,具有部分重组活性的特定重组激活基因 1 变体蛋白可能产生 Omenn 综合征或常见可变免疫缺陷表型。在包括常见可变免疫缺陷和 Wiskott-Aldrich 综合征在内的几种原发性免疫缺陷患者中,研究了中枢和外周 B 细胞耐受,以解释自身免疫和炎症性疾病的发生。报道了 IL-12 和 IL-15 在增强自然杀伤细胞活性中的作用。更多的州正在实施 T 细胞缺陷的新生儿筛查,并正在实现其定义严重联合免疫缺陷的真实发病率和为这些患者提供最佳生存机会的早期治疗的目标。报道了造血干细胞移植和基因治疗对严重免疫缺陷的确定性治疗取得了成功,并越来越明确了获得最佳结果所需的条件。HIV 感染的进展方向是开发有效的疫苗和消除隐藏的潜伏病毒库。