Arthrogryposis refers to congenital contracture in at least two different body parts. When distal joints are primarily involved, the term distal arthrogryposis (DA) is used. The recognition of clinically distinct subtypes of DA has proven very useful in mapping the disease genes for this genetically heterogeneous condition. DA5D is characterized by ocular involvement usually in the form of ptosis and incomitant strabismus, but extraocular manifestations have also been reported. In a multiplex consanguineous family with DA5D, we combined autozygosity mapping and exome sequencing to identify a novel mutation in ECEL1. This was followed by targeted sequencing of this gene in another two extended consanguineous family with the same phenotype, which revealed two additional novel homozygous mutations. Our results support the recent identification of mutations in ECEL1 as a disease gene in DA5D and expand the clinical and allelic spectrum of this condition.

译文

甲状旁腺病是指至少两个不同身体部位的先天性挛缩。当主要涉及远端关节时,将使用术语远端关节固定术(DA)。事实证明,DA的临床独特亚型的识别对于针对这种遗传异质性疾病的疾病基因作图非常有用。 DA5D的特点是眼部受累通常以上睑下垂和斜视性斜视的形式出现,但也有眼外表现的报道。在具有DA5D的多重血缘家族中,我们结合了纯合子作图和外显子组测序来鉴定ECEL1中的新突变。然后在具有相同表型的另外两个近亲家族中对该基因进行靶向测序,这揭示了另外两个新的纯合突变。我们的结果支持了最近鉴定出DA5D中ECEL1作为疾病基因的突变,并扩大了这种疾病的临床和等位基因谱。

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