Alisch Franz, Weichert Alexander, Kalache Karim, Paradiso Viola, Longardt Ann Carolin, Dame Christof, Hoffmann Katrin, Horn Denise
Institut für Medizinische Genetik und Humangenetik, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Klinik für Geburtsmedizin, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Am J Med Genet A. 2017 Jan;173(1):254-259. doi: 10.1002/ajmg.a.37997. Epub 2016 Oct 7.
Gordon syndrome or distal arthrogryposis type 3 is a rare autosomal dominant disorder characterized by contractures of upper and lower limbs. It is distinguishable from other forms of distal arthrogryposis by cleft palate and short stature. Recently, Gordon syndrome has been associated to heterozygous mutations in the piezo-type mechanosensitive ion channel component 2 gene (PIEZO2). Different mutations of this gene also cause distal arthrogryposis type 5 and Marden-Walker syndrome. Dysfunction of this ion channel provides pleiotropic effects on joints, ocular muscles, and bone development. Here, we present a family with three affected individuals exhibiting multiple contractures (metacarpo-phalangeal and interphalangeal joints as well as elbow, shoulder, knee, and ankle joints), clubfeet, short stature, bifid uvula/cleft palate, and a distinct facial phenotype including ptosis. In addition, mild intellectual disability and delay in psychomotor development are obvious. The multigenerational phenotypic spectrum of Gordon syndrome is present in the 37-year-old father, his 4-year-old son and a male neonate showing typical signs of arthrogryposis in the prenatal ultrasound examination already seen at 13 week of gestation. In all affected family members, we identified the PIEZO2 mutation c.8057G>A (p.Arg2686His) by Sanger sequencing. Our analysis indicated that mild delay in psychomotor development and intellectual disability could be part of the phenotypic spectrum of Gordon syndrome. © 2016 Wiley Periodicals, Inc.
戈登综合征或3型远端关节挛缩症是一种罕见的常染色体显性疾病,其特征为上下肢挛缩。它通过腭裂和身材矮小与其他形式的远端关节挛缩症相区分。最近,戈登综合征已与压电型机械敏感离子通道成分2基因(PIEZO2)的杂合突变相关联。该基因的不同突变还会导致5型远端关节挛缩症和马登 - 沃克综合征。这种离子通道功能障碍对关节、眼肌和骨骼发育具有多效性影响。在此,我们报告一个有三名患者的家系,这些患者表现出多处挛缩(掌指关节和指间关节以及肘、肩、膝和踝关节)、马蹄内翻足、身材矮小、悬雍垂裂/腭裂,以及包括上睑下垂在内的独特面部表型。此外,轻度智力残疾和精神运动发育迟缓也很明显。戈登综合征的多代表型谱出现在37岁的父亲、他4岁的儿子以及一名男性新生儿身上,该新生儿在妊娠13周时的产前超声检查中已显示出典型的关节挛缩体征。在所有受影响的家庭成员中,我们通过桑格测序鉴定出PIEZO2突变c.8057G>A(p.Arg2686His)。我们的分析表明,精神运动发育轻度迟缓及智力残疾可能是戈登综合征表型谱的一部分。© 2016威利期刊公司