Caspary T, Cleary M A, Perlman E J, Zhang P, Elledge S J, Tilghman S M
Howard Hughes Medical Institute, Princeton University, Princeton, New Jersey 08544, USA.
Genes Dev. 1999 Dec 1;13(23):3115-24. doi: 10.1101/gad.13.23.3115.
Beckwith-Wiedemann syndrome (BWS) is a clinically variable disorder characterized by somatic overgrowth, macroglossia, abdominal wall defects, visceromegaly, and an increased susceptibility to childhood tumors. The disease has been linked to a large cluster of imprinted genes at human chromosome 11p15.5. A subset of BWS patients has been identified with loss-of-function mutations in p57(KIP2), a maternally expressed gene encoding a G(1) cyclin-dependent kinase inhibitor. Some patients display loss of imprinting of IGF2, a fetal-specific growth factor that is paternally expressed. To understand how the same disease can result from misregulation of two linked, but unrelated, genes, we generated a mouse model for BWS that both harbors a null mutation in p57(Kip2) and displays loss of Igf2 imprinting. These mice display many of the characteristics of BWS, including placentomegaly and dysplasia, kidney dysplasia, macroglossia, cleft palate, omphalocele, and polydactyly. Some, but not all, of the phenotypes are shown to be Igf2 dependent. In two affected tissues, the two imprinted genes appear to act in an antagonistic manner, a finding that may help explain how BWS can arise from mutations in either gene.
贝克威思-维德曼综合征(BWS)是一种临床症状多样的疾病,其特征为躯体过度生长、巨舌、腹壁缺损、内脏肿大以及儿童期肿瘤易感性增加。该疾病与人类染色体11p15.5上的一大簇印记基因有关。已鉴定出一部分BWS患者在p57(KIP2)基因存在功能丧失突变,p57(KIP2)是一个母系表达的基因,编码一种G1期细胞周期蛋白依赖性激酶抑制剂。一些患者表现出胰岛素样生长因子2(IGF2)印记缺失,IGF2是一种父系表达的胎儿特异性生长因子。为了解同一疾病如何由两个相关但不相关的基因调控异常导致,我们构建了一个BWS小鼠模型,该模型既携带p57(Kip2)基因的无效突变,又表现出Igf2印记缺失。这些小鼠表现出许多BWS的特征,包括胎盘肿大和发育异常、肾发育异常、巨舌、腭裂、脐膨出和多指(趾)畸形。部分(而非全部)表型显示为Igf2依赖性。在两个受影响的组织中,这两个印记基因似乎以拮抗方式起作用,这一发现可能有助于解释BWS如何由任一基因的突变引起。