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史密斯-马吉尼斯综合征:7例巴西患者的临床评估

Smith-Magenis syndrome: clinical evaluation in seven Brazilian patients.

作者信息

Gamba B F, Vieira G H, Souza D H, Monteiro F F, Lorenzini J J, Carvalho D R, Morreti-Ferreira D

机构信息

Departamento de Genética, Instituto de Biociências, Universidade Estadual Paulista Julio de Mesquita Filho, Botucatu, SP, Brasil.

出版信息

Genet Mol Res. 2011 Oct 31;10(4):2664-70. doi: 10.4238/2011.October.31.17.

Abstract

Smith-Magenis syndrome (SMS) is a complex congenital anomaly characterized by craniofacial anomalies, neurological and behavioral disorders. SMS is caused by a deletion in region 17p11.2, which includes the RAI1 gene (90% of cases), or by point mutation in the RAI1 gene (10% of cases). Laboratory diagnosis is through cytogenetic analysis by GTG banding and molecular cytogenetic analysis by FISH. We carried out an active search for patients in Associations of Parents and Friends of Exceptional Children (APAE) of São Paulo and genetic centers in Brazil. Forty-eight patients were screened for mental retardation, craniofacial abnormalities and stereotyped behavior with a diagnosis of SMS. In seven of them, chromosome banding at high resolution demonstrated chromosome 17p11.2 deletions, confirmed by FISH. We also made a meta-analysis of 165 cases reported between 1982 and 2010 to compare with the clinical data of our sample. We demonstrated differences between the frequencies of clinical signs among the cases reported and seven Brazilian cases of this study, such as dental anomalies, strabismus, ear infections, deep hoarse voice, hearing loss, and cardiac defects. Although the gold standard for diagnosis of SMS is FISH, we found that the GTG banding technique developed to evaluate chromosome 17 can be used for the SMS diagnosis in areas where the FISH technique is not available.

摘要

史密斯-马吉尼斯综合征(SMS)是一种复杂的先天性异常疾病,其特征为颅面异常、神经和行为障碍。SMS由17p11.2区域的缺失引起(90%的病例),该区域包含RAI1基因,或由RAI1基因的点突变引起(10%的病例)。实验室诊断通过GTG显带的细胞遗传学分析和FISH的分子细胞遗传学分析进行。我们在圣保罗的特殊儿童家长和朋友协会(APAE)以及巴西的遗传中心积极寻找患者。对48名患有智力障碍、颅面异常和刻板行为的患者进行了筛查,诊断为SMS。其中7例经高分辨率染色体显带显示17号染色体p11.2缺失,并经FISH证实。我们还对1982年至2010年期间报告的165例病例进行了荟萃分析,以与我们样本的临床数据进行比较。我们展示了报告病例与本研究的7例巴西病例之间临床体征频率的差异,如牙齿异常、斜视、耳部感染、声音低沉沙哑、听力损失和心脏缺陷。尽管诊断SMS的金标准是FISH,但我们发现,为评估17号染色体而开发的GTG显带技术可用于无法使用FISH技术的地区的SMS诊断。

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