Brady Angela F, Demirdas Serwet, Fournel-Gigleux Sylvie, Ghali Neeti, Giunta Cecilia, Kapferer-Seebacher Ines, Kosho Tomoki, Mendoza-Londono Roberto, Pope Michael F, Rohrbach Marianne, Van Damme Tim, Vandersteen Anthony, van Mourik Caroline, Voermans Nicol, Zschocke Johannes, Malfait Fransiska
Am J Med Genet C Semin Med Genet. 2017 Mar;175(1):70-115. doi: 10.1002/ajmg.c.31550.
The Ehlers-Danlos syndromes comprise a clinically and genetically heterogeneous group of heritable connective tissue disorders, which are characterized by joint hypermobility, skin hyperextensibility, and tissue friability. In the Villefranche Nosology, six subtypes were recognized: The classical, hypermobile, vascular, kyphoscoliotic, arthrochalasis, and dermatosparaxis subtypes of EDS. Except for the hypermobile subtype, defects had been identified in fibrillar collagens or in collagen-modifying enzymes. Since 1997, a whole spectrum of novel, clinically overlapping, rare EDS-variants have been delineated and genetic defects have been identified in an array of other extracellular matrix genes. Advances in molecular testing have made it possible to now identify the causative mutation for many patients presenting these phenotypes. The aim of this literature review is to summarize the current knowledge on the rare EDS subtypes and highlight areas for future research. © 2017 Wiley Periodicals, Inc.
埃勒斯-当洛综合征是一组临床和遗传异质性的遗传性结缔组织疾病,其特征为关节活动过度、皮肤过度伸展和组织脆弱性。在维勒弗朗什分类法中,确认了六种亚型:经典型、活动过度型、血管型、脊柱后侧凸型、关节松弛型和皮肤松弛型埃勒斯-当洛综合征。除活动过度型外,已在纤维状胶原蛋白或胶原蛋白修饰酶中发现缺陷。自1997年以来,已经确定了一系列新的、临床症状重叠的罕见埃勒斯-当洛综合征变体,并且在一系列其他细胞外基质基因中发现了遗传缺陷。分子检测技术的进步使得现在能够为许多表现出这些表型的患者鉴定致病突变。这篇文献综述的目的是总结关于罕见埃勒斯-当洛综合征亚型的当前知识,并突出未来研究的领域。© 2017威利期刊公司