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11例日本综合征性颅缝早闭患者FGFR1-3的突变分析。

Mutation analysis of FGFR1-3 in 11 Japanese patients with syndromic craniosynostoses.

作者信息

Ohishi Akira, Nishimura Gen, Kato Fumiko, Ono Hiroyuki, Maruwaka Kaori, Ago Mako, Suzumura Hiroshi, Hirose Etsuko, Uchida Yuki, Fukami Maki, Ogata Tsutomu

机构信息

Department of Regional Neonatal-Perinatal Medicine, Hamamatsu University School of Medicine, Hamamatsu, Japan.

Department of Pediatrics, Hamamatsu University School of Medicine, Hamamatsu, Japan.

出版信息

Am J Med Genet A. 2017 Jan;173(1):157-162. doi: 10.1002/ajmg.a.37992. Epub 2016 Sep 28.

Abstract

Syndromic craniosynostoses usually occur as single gene disorders. In this study, we analyzed FGFR1-3 genes in four patients with Crouzon syndrome (CS), four patients with Pfeiffer syndrome type 2 (PS-2), one patient with Jackson-Weiss syndrome (JWS), and two patients (sisters) with Muenke syndrome (MS). FGFR2 and FGFR3 mutations were identified in 10 of the 11 patients. Notably, we found a novel FGFR2 p.Asn549Thr mutation in a patient with CS, and a novel FGFR2 p.Ser347Cys mutation in a patient with JWS (thus, this patient was turned out to have an FGFR2-related PS-variant). We also identified an FGFR2 p.Ser252Leu mutation in a phenotypically normal father of a daughter with CS, and an FGFR3 p.Pro250Arg mutation in a mildly macrocephalic father of sisters with MS. These findings, together with previous data, imply that the same FGFR2 mutations can be associated with a wide range of phenotypes including clinically different forms of syndromic craniosynostosis and apparently normal phenotype, depending on other (epi)genetic and environmental factors. Thus, genetic studies are recommended not only for obviously affected individuals but also for family members with apparently normal phenotype or non-specific subtle abnormal phenotype, to allow for pertinent genetic counseling. © 2016 Wiley Periodicals, Inc.

摘要

综合征性颅缝早闭通常表现为单基因疾病。在本研究中,我们分析了4例患有克鲁宗综合征(CS)、4例患有2型 Pfeiffer 综合征(PS-2)、1例患有杰克逊-韦斯综合征(JWS)以及2例(姐妹)患有蒙克综合征(MS)患者的FGFR1-3基因。在11例患者中的10例中鉴定出FGFR2和FGFR3突变。值得注意的是,我们在1例CS患者中发现了一种新的FGFR2 p.Asn549Thr突变,在1例JWS患者中发现了一种新的FGFR2 p.Ser347Cys突变(因此,该患者被证明患有FGFR2相关的PS变异型)。我们还在1例CS女儿的表型正常的父亲中鉴定出FGFR2 p.Ser252Leu突变,在1例MS姐妹的轻度巨头畸形父亲中鉴定出FGFR3 p.Pro250Arg突变。这些发现与先前的数据一起表明,相同的FGFR2突变可与广泛的表型相关,包括临床上不同形式的综合征性颅缝早闭和明显正常的表型,这取决于其他(表观)遗传和环境因素。因此,建议不仅对明显受影响的个体进行基因研究,而且对表型明显正常或具有非特异性轻微异常表型的家庭成员进行基因研究,以便进行相关的遗传咨询。© 2016威利期刊公司

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