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[威廉姆斯-贝伦综合征:多学科诊疗方法]

[Williams-Beuren syndrome: a multidisciplinary approach].

作者信息

Lacroix A, Pezet M, Capel A, Bonnet D, Hennequin M, Jacob M-P, Bricca G, Couet D, Faury G, Bernicot J, Gilbert-Dussardier B

机构信息

Laboratoire langage, mémoire et développement cognitif, CNRS, UMR 6215, 99, avenue du Recteur-Pineau, 86000 Poitiers, France.

出版信息

Arch Pediatr. 2009 Mar;16(3):273-82. doi: 10.1016/j.arcped.2008.11.011. Epub 2008 Dec 18.

Abstract

Williams-Beuren syndrome (WBS) (OMIM# 194050) is a rare, most often sporadic, genetic disease caused by a chromosomal microdeletion at locus 7q11.23 involving 28 genes. Among these, the elastin gene codes for the essential component of the arterial extracellular matrix. Developmental disorders usually associate an atypical face, cardiovascular malformations (most often supravalvular aortic stenosis and/or pulmonary artery stenosis) and a unique neuropsychological profile. This profile is defined by moderate mental retardation, relatively well-preserved language skills, visuospatial deficits and hypersociability. Other less known or rarer features, such as neonatal hypercalcemia, nutrition problems in infancy, ophthalmological anomalies, hypothyroidism, growth retardation, joint disturbances, dental anomalies and hypertension arising in adolescence or adulthood, should be treated. The aim of this paper is to summarize the major points of WBS regarding: (i) the different genes involved in the deletion and their function, especially the elastin gene and recent reports of rare forms of partial WBS or of an opposite syndrome stemming from a microduplication of the 7q11.23 locus, (ii) the clinical features in children and adults with a focus on cardiovascular injury, and (iii) the specific neuropsychological profile of people with WBS through its characteristics, the brain structures involved, and learning.

摘要

威廉姆斯-贝伦综合征(WBS)(OMIM编号:194050)是一种罕见的、多为散发性的遗传性疾病,由7号染色体长臂11.23位点的染色体微缺失导致,该缺失涉及28个基因。其中,弹性蛋白基因编码动脉细胞外基质的重要成分。发育障碍通常伴有非典型面容、心血管畸形(最常见的是主动脉瓣上狭窄和/或肺动脉狭窄)以及独特的神经心理学特征。这种特征表现为中度智力障碍、语言能力相对保留较好、视觉空间缺陷和过度社交性。其他不太为人所知或较为罕见的特征,如新生儿高钙血症、婴儿期营养问题、眼科异常、甲状腺功能减退、生长发育迟缓、关节紊乱、牙齿异常以及青春期或成年期出现的高血压等,都应予以治疗。本文旨在总结威廉姆斯-贝伦综合征在以下方面的要点:(i)与缺失相关的不同基因及其功能,特别是弹性蛋白基因,以及近期关于部分威廉姆斯-贝伦综合征罕见形式或由7号染色体长臂11.23位点微重复导致的相反综合征的报道;(ii)儿童和成人的临床特征,重点关注心血管损伤;(iii)通过其特征、涉及的脑结构和学习情况来阐述威廉姆斯-贝伦综合征患者的特定神经心理学特征。

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[Williams-Beuren syndrome: a multidisciplinary approach].[威廉姆斯-贝伦综合征:多学科诊疗方法]
Arch Pediatr. 2009 Mar;16(3):273-82. doi: 10.1016/j.arcped.2008.11.011. Epub 2008 Dec 18.

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