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两名携带 CDT1 双等位基因突变的 Meier-Gorlin 综合征成人患者成功妊娠。

Successful pregnancies in an adult with Meier-Gorlin syndrome harboring biallelic CDT1 variants.

机构信息

Department of Pathology, Dunedin School of Medicine, University of Otago, Dunedin, New Zealand.

MRC Human Genetics Unit, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK.

出版信息

Am J Med Genet A. 2021 Mar;185(3):871-876. doi: 10.1002/ajmg.a.62016. Epub 2020 Dec 18.

Abstract

Meier-Gorlin syndrome is an autosomal recessively inherited disorder of growth retardation, accompanied by microtia and patellae a/hypoplasia and characteristic facies. Pathogenic variants in genes associated with the initiation of DNA replication underlie the condition, with biallelic variants in CDT1 the most common cause. Using 10× Chromium genome sequencing, we report CDT1 variants in an adult female, with an inframe amino acid deletion inherited in trans with a deep intronic variant which likely serves as the branchpoint site in Intron 8. Splicing defects arising from this variant were confirmed through in vitro analysis. At 49 years, she represents the oldest patient with a molecular diagnosis described in the literature and is the first reported patient with Meier-Gorlin syndrome to have carried a successful pregnancy to term. Both of her pregnancies were complicated by postpartum hemorrhage and upon subsequent necessary hysterectomy, revealed uterine abnormalities. There is scant knowledge on reproductive ability and success in patients with Meier-Gorlin syndrome. Successful pregnancies among other clinically recognizable forms of primordial dwarfism have also not been described previously. This case is therefore of clinical interest for many forms of inherited growth retardation, and will assist in providing more information and clinical guidance for females of reproductive age.

摘要

Meier-Gorlin 综合征是一种常染色体隐性遗传的生长发育迟缓疾病,伴有小耳畸形和髌骨发育不良及特征性面容。该疾病的病因是与 DNA 复制起始相关的基因中的致病性变异,双等位基因变异在 CDT1 中最为常见。我们使用 10× Chromium 基因组测序,报告了一名成年女性的 CDT1 变异,该变异为反式遗传的框移缺失,与 8 号内含子中的深内含子变异相关,该变异可能作为分支点。通过体外分析证实了该变异导致的剪接缺陷。该患者 49 岁,是文献中描述的患有分子诊断的最年长患者,也是首例报告的携带 Meier-Gorlin 综合征并成功足月妊娠的患者。她的两次妊娠均并发产后出血,随后进行的必要子宫切除术显示存在子宫异常。对于 Meier-Gorlin 综合征患者的生育能力和成功率知之甚少。其他临床上可识别的原始侏儒症形式也没有成功妊娠的报道。因此,对于多种形式的遗传性生长发育迟缓,该病例具有重要的临床意义,并将有助于为育龄女性提供更多的信息和临床指导。

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