Shotelersuk Vorasuk, Srichomthong Chalurmpon, Yoshiura Koh-ichiro, Niikawa Norio
Section on Medical Genetics and Metabolism, Department of Pediatrics, King Chulalongkorn Memorial Hospital, Bangkok 10330, Thailand.
Int J Mol Med. 2003 Apr;11(4):505-7.
Van der Woude syndrome (VWS) is an autosomal dominant disorder and the most common cleft syndrome characterized by cleft lip and palate with lip pits. Very recently, mutations in the interferon regulatory factor 6 gene (IRF6) were identified to cause VWS in patients of northern European descent. We describe a Thai family with VWS. The proband, an 8-month-old boy, had bilateral complete cleft lip and palate, and two conical elevations with lip pits on his lower lip. Four other family members had various manifestations of the clefts and lower lip pits. Mutation analysis of the proband and his mother for the entire coding region of IRF6 identified a novel mutation, 1234del(C), in its exon 9. The deletion is expected to result in some amino acid changes followed by truncation at amino acid 435. This observation supports that IRF6 is the gene responsible for VWS across different populations and that haploinsufficiency of the gene disturbs development of the lip and palate.
范德伍德综合征(VWS)是一种常染色体显性疾病,是最常见的腭裂综合征,其特征为唇腭裂伴唇凹。最近,在北欧裔患者中发现干扰素调节因子6基因(IRF6)的突变可导致VWS。我们描述了一个患有VWS的泰裔家庭。先证者是一名8个月大的男孩,患有双侧完全性唇腭裂,下唇有两个带唇凹的圆锥形隆起。其他四名家庭成员有不同程度的腭裂和下唇凹表现。对先证者及其母亲的IRF6整个编码区进行突变分析,在其外显子9中发现了一个新的突变1234del(C)。预计该缺失会导致一些氨基酸变化,随后在第435位氨基酸处截短。这一观察结果支持IRF6是不同人群中导致VWS的基因,且该基因的单倍剂量不足会干扰唇和腭的发育。
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