Jiang Lei, Liu Chao, Wang Wei-qing, Ye Lei, Zhu Na, Zhou Wei-wei, Su Ting-wei, Li Xiao-ying, Ning Guang
Shanghai Clinical Center for Endocrine and Metabolic Diseases, Department of Endocrinology and Metabolism, Ruijin Hospital, Shanghai Jiaotong University School of Medicine, Shanghai 200025, China.
Zhonghua Nei Ke Za Zhi. 2006 Sep;45(9):730-3.
Leprechaunism is an inherited insulin resistance syndrome, caused by homozygous or compound-heterozygous mutations in the insulin receptor gene (INSR). Clinical and molecular genetic research was carried out in one 17-year old girl with leprechaunism and her family members.
History and laboratory tests were routinely taken. DNA samples were obtained from the proband and 4 of the family members. PCR was done on all exons of INSR and the products of PCR were sequenced directly.
The proband had the following features: apparent cessation of growth, elfin-like face, emaciation, hirsutism and acanthosis nigricans. She had hyperglycemia (fasting blood glucose 15.8 mmol/L; glycosylated forms of hemoglobin 12%) and it was resistant to the treatment of insulin. She was found to have W659R mutation at 9 exon and V1054M mutation at 17 exon of INSR as heterozygotes. This compound mutation is a newly found type. For her father there was only V1054M mutation and her mother only W659R mutation. No mutation was identified in her sister.
The patient was diagnosed as leprechaunism according to her clinical presentations and biochemical examinations. The new transition mutation W659R at 9 exon and V1054M at 17 exon in INSR is the pathologic cause in this patient with leprechaunism.
矮妖精貌综合征是一种遗传性胰岛素抵抗综合征,由胰岛素受体基因(INSR)的纯合或复合杂合突变引起。对一名患有矮妖精貌综合征的17岁女孩及其家庭成员进行了临床和分子遗传学研究。
常规采集病史和进行实验室检查。从先证者及其4名家庭成员获取DNA样本。对INSR的所有外显子进行聚合酶链反应(PCR),并直接对PCR产物进行测序。
先证者有以下特征:生长明显停滞、小精灵样面容、消瘦、多毛和黑棘皮症。她患有高血糖(空腹血糖15.8 mmol/L;糖化血红蛋白12%),且对胰岛素治疗耐药。发现她INSR的第9外显子有W659R突变,第17外显子有V1054M突变,均为杂合子。这种复合突变是新发现的类型。她的父亲只有V1054M突变,母亲只有W659R突变。其妹妹未发现突变。
根据临床表现和生化检查,该患者被诊断为矮妖精貌综合征。INSR第9外显子新的转换突变W659R和第17外显子的V1054M是该矮妖精貌综合征患者的致病原因。