Sangu Noriko, Shimosato Tsuyoshi, Inoda Hirosato, Shimada Shino, Shimojima Keiko, Ando Tomohiro, Yamamoto Toshiyuki
Department of Oral and Maxillofacial Surgery, School of Medicine, Tokyo Women's Medical University, Tokyo; Institute for Integrated Medical Sciences, Tokyo Women's Medical University, Tokyo.
Congenit Anom (Kyoto). 2013 Dec;53(4):166-9. doi: 10.1111/cga.12013.
Cherubism is a rare genetic disorder characterized by progressive facial deformity caused by non-neoplastic bone lesions in the mandible and/or the maxilla. Src homology-3 binding protein 2 gene (SH3BP2) has been found to be the responsible gene, with alterations in six amino acids noted in patients with this condition. Recently, mutations in this domain have been found to cause stabilization of SH3BP2 by uncoupling with tankyrase. In this study, we identified a new 2-bp mutation that led to a recurrent amino acid change in a sporadic case of cherubism. Our findings indicate that it is important to understand the pattern of progress in typical cherubism.
cherubism是一种罕见的遗传性疾病,其特征是下颌骨和/或上颌骨的非肿瘤性骨病变导致进行性面部畸形。Src同源3结合蛋白2基因(SH3BP2)已被发现是致病基因,患有这种疾病的患者中发现有六个氨基酸发生改变。最近,已发现该结构域中的突变通过与端粒酶解偶联导致SH3BP2稳定。在本研究中,我们在一例散发性cherubism病例中鉴定出一个新的2碱基突变,该突变导致了反复出现的氨基酸变化。我们的研究结果表明,了解典型cherubism的进展模式很重要。