Hiraki Yoko, Miyatake Satoko, Hayashidani Michiko, Nishimura Yutaka, Matsuura Hiroo, Kamada Masahiro, Kawagoe Takuji, Yunoki Keiji, Okamoto Nobuhiko, Yofune Hiroko, Nakashima Mitsuko, Tsurusaki Yoshinori, Satisu Hirotomo, Murakami Akira, Miyake Noriko, Nishimura Gen, Matsumoto Naomichi
Hiroshima Municipal Center for Child Health and Development, Hiroshima, Japan.
Am J Med Genet A. 2014 Jan;164A(1):231-6. doi: 10.1002/ajmg.a.36228. Epub 2013 Nov 25.
Cantu syndrome is an autosomal dominant overgrowth syndrome associated with facial dysmorphism, congenital hypertrichosis, and cardiomegaly. Some affected individuals show bone undermodeling of variable severity. Recent investigations revealed that the disorder is caused by a mutation in ABCC9, encoding a regulatory SUR2 subunit of an ATP-sensitive potassium channel mainly expressed in cardiac and skeletal muscle as well as vascular smooth muscle. We report here on a Japanese family with this syndrome. An affected boy and his father had a novel missense mutation in ABCC9. Each patient had a coarse face and hypertrichosis. However, cardiomegaly was seen only in the boy, and macrosomia only in the father. Skeletal changes were not evident in either patient. Craniosynostosis in the boy and the development of aortic aneurysm in the father are previously undescribed associations with Cantu syndrome.
坎图综合征是一种常染色体显性过度生长综合征,与面部畸形、先天性多毛症和心脏肥大有关。一些受影响的个体表现出不同严重程度的骨骼发育不足。最近的研究表明,该疾病是由ABCC9基因突变引起的,ABCC9编码一种ATP敏感性钾通道的调节性SUR2亚基,主要在心肌、骨骼肌以及血管平滑肌中表达。我们在此报告一个患有该综合征的日本家庭。一名患病男孩及其父亲在ABCC9基因中存在一种新的错义突变。每位患者都有面容粗糙和多毛症。然而,心脏肥大仅在男孩中出现,巨大儿仅在父亲中出现。两名患者均未出现明显的骨骼变化。男孩的颅缝早闭和父亲的主动脉瘤形成是先前未描述的与坎图综合征相关的情况。