Takeda K, Sugiura T
Clinical Laboratory Medicine, Kochi Medical School, Nankoku.
Rinsho Byori. 2000 May;48(5):396-408.
The biological effects of hormones are mediated by plasma membrane and nuclear receptors which transmit extracellular signals to the cytoplasm and nucleus. The mutations in these receptors can affect normal signal transduction with loss-of-function mutations leading to hormone resistance and gain-of-function mutations leading to constitutive activation of signaling pathways. Mutations in plasma membrane are involved in a large number of clinical disorders, including dwarfism, Laron syndrome, congenital hyperthyroidism or hypothyroidism, nephrogenic diabetes insipidus, resistance to TSH, LH, FSH and ACTH, Jansen's metaphyseal chondrodysplasia and autosomal dominant hypoparathyroidism. While, mutations in nuclear receptors are the cause of resistance to glucocorticoid, androgen, estrogen, thyroid hormone and vitamin D. The loss-of-function mutations leading to familial hormone resistance disorders are germ line in origin whereas the gain-of-function mutations leading to constitutively active receptors are somatic. The plasma membrane receptors for those disorders except GH resistance consist of seven spanning-transmembranes which couple with GTP binding (G) protein. Abnormal G protein also exhibits gain and loss of function for hormones, leading to tumors and pseudo-hypoparathyroidism, respectively. This review summarizes molecular defects in hormone receptors and G proteins and their associated clinical features.
激素的生物学效应由质膜和核受体介导,这些受体将细胞外信号传递至细胞质和细胞核。这些受体中的突变可影响正常信号转导,功能丧失性突变导致激素抵抗,功能获得性突变导致信号通路的组成性激活。质膜突变涉及大量临床疾病,包括侏儒症、拉伦综合征、先天性甲状腺功能亢进或减退、肾性尿崩症、对促甲状腺激素、促黄体生成素、促卵泡生成素和促肾上腺皮质激素的抵抗、詹森干骺端软骨发育不良和常染色体显性遗传性甲状旁腺功能减退。而核受体突变是导致对糖皮质激素、雄激素、雌激素、甲状腺激素和维生素D抵抗的原因。导致家族性激素抵抗疾病的功能丧失性突变起源于生殖细胞,而导致组成性激活受体的功能获得性突变是体细胞性的。除生长激素抵抗外,那些疾病的质膜受体由七个跨膜结构域组成,它们与GTP结合(G)蛋白偶联。异常的G蛋白对激素也表现出功能获得和功能丧失,分别导致肿瘤和假性甲状旁腺功能减退。本综述总结了激素受体和G蛋白的分子缺陷及其相关临床特征。