Cho Hyun-Jung, Sung Duk-Hyun, Ki Chang-Seok
Department of Laboratory Medicine and Genetics, Samsung Medical Center, Sungkyunkwan University School of Medicine, 50 Irwon-dong Gangnam-gu, Seoul, 135-710 Korea.
Muscle Nerve. 2007 Sep;36(3):384-6. doi: 10.1002/mus.20792.
Mutations in the Berardinelli-Seip congenital lipodystrophy (BSCL2) gene have been identified in families with distal hereditary motor neuropathy (dHMN) and in families with SPG17-linked Silver syndrome. We studied the first Korean families with clinical features resembling classic Silver syndrome and dHMN type V. Direct sequencing analysis of the BSCL2 gene revealed a Ser90Leu mutation in the proband, a younger sister, and one of two sons of the proband. The clinical patterns in this family include presentation with lower-limb and hand-muscle involvement early in the disease course as well as the presence of Babinski signs with nonprogressive mild spastic paraparesis, resembling classic Silver syndrome and dHMN type V. This study reaffirms the clinical phenotype of the disorders associated with a BSCL2 Ser90Leu mutation and describes a genetically proven family with Silver syndrome and dHMN type V in Asia.
在患有远端遗传性运动神经病(dHMN)的家族以及患有SPG17连锁银综合征的家族中,已发现贝拉尔迪内利-塞普先天性脂肪营养不良(BSCL2)基因的突变。我们研究了首例具有类似经典银综合征和V型dHMN临床特征的韩裔家族。对BSCL2基因进行直接测序分析发现,先证者、其妹妹以及先证者两个儿子中的一个存在Ser90Leu突变。该家族的临床模式包括在病程早期出现下肢和手部肌肉受累,以及伴有非进行性轻度痉挛性截瘫的巴宾斯基征,类似于经典银综合征和V型dHMN。本研究再次证实了与BSCL2 Ser90Leu突变相关疾病的临床表型,并描述了亚洲一个经基因证实的银综合征和V型dHMN家族。