Department of Human Genetics, National Center for Global Health and Medicine, Tokyo, Japan.
Department of Medical Genetics, Shinshu University School of Medicine, Matsumoto, Japan.
Adv Exp Med Biol. 2021;1348:235-249. doi: 10.1007/978-3-030-80614-9_10.
Ehlers-Danlos syndrome (EDS) is a genetically and clinically heterogeneous group of connective tissue disorders that typically present with skin hyperextensibility, joint hypermobility, and tissue fragility. The major cause of EDS appears to be impaired biosynthesis and enzymatic modification of collagen. In this chapter, we discuss two types of EDS that are associated with proteoglycan abnormalities: spondylodysplastic EDS and musculocontractural EDS. Spondylodysplastic EDS is caused by pathogenic variants in B4GALT7 or B3GALT6, both of which encode key enzymes that initiate glycosaminoglycan synthesis. Musculocontractural EDS is caused by mutations in CHST14 or DSE, both of which encode enzymes responsible for the post-translational biosynthesis of dermatan sulfate. The clinical and molecular characteristics of both types of EDS are described in this chapter.
埃勒斯-当洛斯综合征(EDS)是一组具有遗传和临床异质性的结缔组织疾病,其特征通常为皮肤过度伸展、关节过度活动和组织脆弱。EDS 的主要原因似乎是胶原蛋白的生物合成和酶修饰受损。在本章中,我们讨论了两种与蛋白聚糖异常相关的 EDS:脊椎发育不良型 EDS 和肌肉收缩型 EDS。脊椎发育不良型 EDS 是由 B4GALT7 或 B3GALT6 的致病性变异引起的,这两种基因均编码起始糖胺聚糖合成的关键酶。肌肉收缩型 EDS 是由 CHST14 或 DSE 的突变引起的,这两种基因均编码负责硫酸皮肤素的翻译后生物合成的酶。本章描述了这两种类型的 EDS 的临床和分子特征。