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一位具有 Phelan-McDermid 综合征典型特征的患者,其高免疫球蛋白 E 水平是由 22q13.2 区域内隐匿性 0.72-Mb 缺失引起的。

A patient with the classic features of Phelan-McDermid syndrome and a high immunoglobulin E level caused by a cryptic interstitial 0.72-Mb deletion in the 22q13.2 region.

机构信息

Department of Genetics, United Laboratories, Tartu University Hospital, Tartu, Estonia; Medical Faculty, University of Tartu, Tartu, Estonia.

出版信息

Am J Med Genet A. 2014 Mar;164A(3):806-9. doi: 10.1002/ajmg.a.36358. Epub 2013 Dec 20.

Abstract

Phelan-McDermid syndrome, also known as the 22q13 deletion syndrome, is a chromosomal microdeletion syndrome characterized by neonatal hypotonia, normal growth, profound developmental delay, absent or delayed speech, and minor dysmorphic features. Almost all of the 22q13 deletions published so far have been described as terminal. It is believed that the SHANK3 gene is the major candidate gene for the neurologic features of the syndrome. Here we describe a patient with a 0.72-Mb interstitial 22q13.2 deletion, intellectual disability, autistic behavior, epilepsy, mild dysmorphic features, and no deletion in the SHANK3 gene. The patient also has urticarial rash and an elevated level of immunoglobulin E, the latter has previously been described only once in a patient with monosomy 22q13.2-qter and SHANK3 gene deletion. To our knowledge, this is one of the smallest interstitial deletion in this region which has been published up to now. Although the patient has the classic phenotype of the 22q13 terminal deletion syndrome, the etiology for the neurologic and immunological features must be due to genes located more proximal to SHANK3 and this is also supported by other previously published cases of interstitial 22q13.2 deletions. The deleted area in our patient is gene-rich (26 genes), containing several known genes with different functions. Two of them-NFAM1 and TNFRSF13C are involved in immune system functioning. We suggest the haploinsufficiency of these genes might be related to hyper IgE syndrome in our patient.

摘要

佩兰-麦克德米德综合征,也称为 22q13 缺失综合征,是一种染色体微缺失综合征,其特征为新生儿低张力、正常生长、严重发育迟缓、言语缺失或延迟、轻微的畸形特征。到目前为止,几乎所有报道的 22q13 缺失都被描述为末端缺失。据信,SHANK3 基因是该综合征神经特征的主要候选基因。在这里,我们描述了一名患者,其存在 0.72Mb 的 22q13.2 片段间缺失,表现为智力障碍、自闭症行为、癫痫、轻度畸形特征,且 SHANK3 基因无缺失。该患者还存在荨麻疹样皮疹和免疫球蛋白 E 水平升高,后者之前仅在 22q13.2-qter 单体性和 SHANK3 基因缺失的患者中描述过一次。据我们所知,这是迄今为止报道的该区域最小的片段间缺失之一。尽管该患者具有 22q13 末端缺失综合征的经典表型,但神经和免疫特征的病因一定是由于位于 SHANK3 更近端的基因引起的,这也得到了其他先前报道的 22q13.2 片段缺失病例的支持。我们患者的缺失区域富含基因(26 个基因),包含几个具有不同功能的已知基因。其中两个-NFAM1 和 TNFRSF13C 参与免疫系统功能。我们提出这些基因的杂合性缺失可能与我们患者的高 IgE 综合征有关。

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