Pitera Jolanta E, Turmaine Mark, Woolf Adrian S, Scambler Peter J
Molecular Medicine Unit, Institute of Child Health, University College London, United Kingdom.
Genesis. 2012 Dec;50(12):892-8. doi: 10.1002/dvg.22045. Epub 2012 Jul 23.
Fraser syndrome (FS) is an autosomal recessive disease characterized by skin lesions and kidney and upper airway malformations. Fraser syndrome 1 (FRAS1) is an extracellular matrix protein, and FRAS1 homozygous mutations occur in some FS individuals. FRAS1 is expressed at the epithelial-mesenchymal interface in embryonic skin and kidney. blebbed mice have a null Fras1 mutation and phenocopy human FS. Like humans with FS, they exhibit a high fetal and neonatal mortality, precluding studies of FRAS1 functions in later life. We generated conditional Fras1 null allele mice. Cre-mediated generalized deletion of this allele generated embryonic skin blisters and renal agenesis characteristic of blebbed mice and human FS. Targeted deletion of Fras1 in kidney podocytes circumvented skin blistering, renal agenesis, and early death. FRAS1 expression was downregulated in maturing glomeruli which then became sclerotic. The data are consistent with the hypothesis that locally produced FRAS1 has roles in glomerular maturation and integrity. This conditional allele will facilitate study of possible role for FRAS1 in other tissues such as the skin.
弗雷泽综合征(FS)是一种常染色体隐性疾病,其特征为皮肤病变以及肾脏和上呼吸道畸形。弗雷泽综合征1(FRAS1)是一种细胞外基质蛋白,部分FS患者存在FRAS1纯合突变。FRAS1在胚胎皮肤和肾脏的上皮 - 间充质界面表达。水泡小鼠存在Fras1无效突变,其表型与人类FS相似。与患有FS的人类一样,它们表现出较高的胎儿和新生儿死亡率,这使得无法对FRAS1在后期生命中的功能进行研究。我们构建了条件性Fras1无效等位基因小鼠。Cre介导的该等位基因的全身性缺失产生了胚胎皮肤水泡和肾脏发育不全,这是水泡小鼠和人类FS的特征。在肾足细胞中靶向缺失Fras1避免了皮肤水泡、肾脏发育不全和早期死亡。在成熟肾小球中FRAS1表达下调,随后肾小球硬化。这些数据与局部产生的FRAS1在肾小球成熟和完整性中起作用的假设一致。这种条件等位基因将有助于研究FRAS1在其他组织如皮肤中的可能作用。