Institute of Medical and Molecular Genetics (INGEMM), Hospital Universitario La Paz, Universidad Autónoma de Madrid, IdiPAZ, Madrid, Spain.
Skeletal dysplasia Multidisciplinary Unit (UMDE) and ERN-BOND, Hospital Universitario La Paz, Madrid, Spain.
Clin Genet. 2021 Feb;99(2):309-312. doi: 10.1111/cge.13876. Epub 2020 Nov 10.
Multiple synostosis syndrome (SYNS) is a heterogeneous group of genetic disorders mainly characterized by multiple joint synostosis due to variants in either NOG, GDF5, FGF9 or GDF6. To date, only two FGF9 variants have been associated with SYNS, characterized with hand and feet joint synostosis and fusion of the elbow and vertebral lumbar joints. Craniosynostosis was also observed in one family. Here, we report the clinical and radiological description of a young girl with a third heterozygous FGF9 variant, NM_002010.2:c.427A>T;p.(Asn143Tyr), which interestingly, is located at the same amino acid as the well characterized spontaneous Eks mouse variant. We also compare the genotype: phenotypes observed between humans and mice with SYNS.
多发性骨融合综合征(SYNS)是一组异质性遗传疾病,主要特征为由于 NOG、GDF5、FGF9 或 GDF6 中的变异导致多个关节骨融合。迄今为止,仅发现两种 FGF9 变异与 SYNS 相关,其特征为手和脚关节骨融合以及肘部和腰椎关节融合。在一个家族中还观察到颅缝早闭。在此,我们报告了一名年轻女孩的临床和影像学描述,她携带第三个杂合性 FGF9 变异,NM_002010.2:c.427A>T;p.(Asn143Tyr),有趣的是,该变异位于与特征明确的自发性 Eks 小鼠变异相同的氨基酸位置。我们还比较了人类和具有 SYNS 的小鼠之间的基因型:表型观察结果。