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进行性骨化性纤维发育不良:临床病程、基因突变及基因型-表型相关性

Fibrodysplasia ossificans progressiva: clinical course, genetic mutations and genotype-phenotype correlation.

作者信息

Hüning Irina, Gillessen-Kaesbach Gabriele

机构信息

Institut für Humangenetik, Universität zu Lübeck, Lübeck, Germany.

出版信息

Mol Syndromol. 2014 Aug;5(5):201-11. doi: 10.1159/000365770. Epub 2014 Aug 7.

Abstract

Fibrodysplasia ossificans progressiva (FOP, MIM 135100) is a rare autosomal dominant genetic disorder and the most disabling condition of heterotopic (extraskeletal) ossification in humans. Mutations in the ACVR1 gene (MIM 102576) were identified as a genetic cause of FOP [Shore et al., 2006]. Most patients with FOP have the same recurrent single nucleotide change c.617G>A, p.R206H in the ACVR1 gene. Furthermore, 11 other mutations in the ACVR1 gene have been described as a cause of FOP. Here, we review phenotypic and molecular findings of 130 cases of FOP reported in the literature from 1982 to April 2014 and discuss possible genotype-phenotype correlations in FOP patients.

摘要

进行性骨化性纤维发育不良(FOP,MIM 135100)是一种罕见的常染色体显性遗传病,也是人类异位(骨骼外)骨化最致残的病症。ACVR1基因(MIM 102576)突变被确定为FOP的遗传病因[Shore等人,2006年]。大多数FOP患者在ACVR1基因中具有相同的复发性单核苷酸变化c.617G>A,p.R206H。此外,ACVR1基因中的其他11种突变也被描述为FOP的病因。在此,我们回顾了1982年至2014年4月文献中报道的130例FOP患者的表型和分子研究结果,并讨论了FOP患者可能的基因型与表型的相关性。

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