摘要
常染色体显性遗传多囊肾病（ADPKD）是最常见的单基因肾病，由PKD1，PKD2突变引起，或者在非常有限的家族中由GANAB基因突变引起。 尽管在过去的20年中已经了解了这种疾病的细胞和分子机制，但具体的治疗方法仍然很少。 实验和临床研究均表明，雷帕霉素（mTOR）途径的哺乳动物或机械靶标在ADPKD的囊肿形成和扩大过程中起重要作用。 在ADPKD的啮齿动物模型中的研究表明，mTOR抑制剂具有显着且持久的肾脏体积减少和肾功能的改善。 在过去的十多年中，研究人员一直致力于在ADPKD患者的临床前研究和临床试验中测试mTOR抑制剂的疗效和安全性。 在这篇综述中，我们将彻底讨论mTOR途径，主要关注当前在理解其在ADPKD中的作用的进展，特别是mTOR抑制剂在临床前研究和临床试验中的最新进展。
关键词: 常染色体显性多囊肾病，mTOR信号，mTOR抑制剂，疗效和安全性，临床前模型，临床试验。
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