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首页> 外文期刊>Proceedings of the National Academy of Sciences of the United States of America >The mTOR pathway is regulated by polycystin-1, and its inhibition reverses renal cystogenesis in polycystic kidney disease
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The mTOR pathway is regulated by polycystin-1, and its inhibition reverses renal cystogenesis in polycystic kidney disease

机译：mTOR途径受多囊藻蛋白-1调节，其抑制作用可逆转多囊肾疾病中的肾脏囊肿发生

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摘要

Autosomal-dominant polycystic kidney disease (ADPKD) is a common genetic disorder that frequently leads to renal failure. Mutations in polycystin-1 (PC1) underlie most cases of ADPKD, but the function of PC1 has remained poorly understood. No preventive treatment for this disease is available. Here, we show that the cytoplasmic tail of PC1 interacts with tuberin, and the mTOR pathway is inappropriately activated in cyst-lining epithelial cells in human ADPKD patients and mouse models. Rapamycin, an inhibitor of mTOR, is highly effective in reducing renal cystogenesis in two independent mouse models of PKD. Treatment of human ADPKD transplant-recipient patients with rapamycin results in a significant reduction in native polycystic kidney size. These results indicate that PC1 has an important function in the regulation of the mTOR pathway and that this pathway provides a target for medical therapy of ADPKD.
机译：常染色体显性多囊肾病（ADPKD）是一种常见的遗传性疾病，经常导致肾功能衰竭。在大多数ADPKD病例中，多囊藻蛋白1（PC1）的突变是基础，但对PC1的功能仍知之甚少。没有针对这种疾病的预防性治疗。在这里，我们显示了PC1的胞质尾与结核菌素相互作用，并且在人类ADPKD患者和小鼠模型的囊肿衬砌上皮细胞中mTOR通路被不适当地激活。雷帕霉素是mTOR的抑制剂，在两种独立的PKD小鼠模型中，在减少肾囊肿生成方面非常有效。雷帕霉素治疗人类ADPKD移植受者患者会导致天然多囊肾大小明显减少。这些结果表明，PC1在mTOR途径的调控中具有重要作用，并且该途径为ADPKD的药物治疗提供了靶点。

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