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胎儿严重胎儿生长受限且母亲患有严重系统性红斑狼疮患者的中央 22q11.2 缺失(LCR22 B-D):CRKL 杂合不足在 22q11.2 缺失综合征发病机制中的进一步证据。

Central 22q11.2 deletion (LCR22 B-D) in a fetus with severe fetal growth restriction and a mother with severe systemic lupus erythematosus: Further evidence of CRKL haploinsufficiency in the pathogenesis of 22q11.2 deletion syndrome.

机构信息

David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, USA.

Division of Maternal Fetal Medicine, Department of Obstetrics and Gynecology, University of California Los Angeles, Los Angeles, California, USA.

出版信息

Am J Med Genet A. 2021 Oct;185(10):3042-3047. doi: 10.1002/ajmg.a.62346. Epub 2021 Jul 1.

DOI:10.1002/ajmg.a.62346
PMID:34196458
Abstract

22q11.2 deletion syndrome (22q11.2 DS, MIM #188400) is the most common chromosomal microdeletion with an incidence of 1 in 4000 live births. 22q11.2 DS patients present with varying penetrance and a broad phenotypic spectrum including dysmorphic features, congenital heart defects, hypoplastic thymus and T-cell deficiency, and hypocalcemia. The typical deletion spans 3 Mb between 4 large blocks of repetitive DNA, known as low copy repeats (LCRs), on chromosome 22 (LCR22) A and D. This deletion is found in ~85% of 22q11.2 DS patients, while only 4-5% have central LCR22B-D (1.5 Mb) and LCR22C-D (0.7 Mb) deletions. We report on a prenatally diagnosed, inherited case of central LCR22B-D 22q11.2 DS, born to a 22-year-old female with multiple autoimmune disorders. These include Sjogren's-syndrome-related antigen A (SSA+) severe systemic lupus erythematosus (SLE) with cutaneous and discoid components and seronegative antiphospholipid syndrome. Amniocentesis was performed due to fetal growth restriction (FGR). FISH with TUPLE1 (HIRA) probe was normal; however, chromosomal microarray identified a ~737 kb heterozygous loss between LCR22B-D. Subsequently, the same deletion was identified in the mother, which included CRKL and 19 other genes but excluded HIRA and TBX1, the typical candidate genes for 22q11.2DS pathogenesis. This case explores how loss of CRKL may contribute to immune dysregulation, as seen in the multiple severe autoimmune phenotypes of the mother, and FGR. Our experience confirms the importance of thorough workup in individuals with reduced penetrance of 22q11.2 DS features or atypical clinical presentations.

摘要

22q11.2 缺失综合征(22q11.2 DS,MIM #188400)是最常见的染色体微缺失,发病率为每 4000 例活产儿中有 1 例。22q11.2 DS 患者的表现具有不同的外显率和广泛的表型谱,包括畸形特征、先天性心脏病、胸腺发育不全和 T 细胞缺陷以及低钙血症。典型的缺失跨越染色体 22 上的 4 个大型重复 DNA 块(称为低拷贝重复序列,LCRs)之间的 3 Mb,即 LCR22A 和 D。该缺失存在于约 85%的 22q11.2 DS 患者中,而只有 4-5%的患者存在中央 LCR22B-D(1.5 Mb)和 LCR22C-D(0.7 Mb)缺失。我们报告了一例产前诊断的遗传性中央 LCR22B-D 22q11.2 DS 病例,该病例的母亲为 22 岁女性,患有多种自身免疫性疾病。这些疾病包括与干燥综合征相关抗原 A(SSA+)相关的严重系统性红斑狼疮(SLE),具有皮肤和盘状成分以及血清阴性抗磷脂综合征。由于胎儿生长受限(FGR)进行了羊膜穿刺术。使用 TUPLE1(HIRA)探针的 FISH 正常;然而,染色体微阵列识别出 LCR22B-D 之间存在约 737 kb 的杂合性缺失。随后,在母亲中也发现了相同的缺失,该缺失包括 CRKL 和 19 个其他基因,但排除了 HIRA 和 TBX1,它们是 22q11.2 DS 发病机制的典型候选基因。该病例探讨了 CRKL 的缺失如何导致免疫失调,正如母亲的多种严重自身免疫表型和 FGR 所见。我们的经验证实了在 22q11.2 DS 特征或非典型临床表现外显率降低的个体中进行彻底检查的重要性。

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