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Review Article

常染色体显性遗传多囊肾病的靶向治疗

卷 26, 期 17, 2019

页: [3081 - 3102] 页: 22

弟呕挨: 10.2174/0929867325666180508095654

价格: $65

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摘要

背景：常染色体显性多囊肾病（ADPKD）是人类最常见的危及生命的遗传性疾病，约有500人受到影响。 ADPKD的特征在于肾脏中的囊肿生长导致进行性实质损伤，并且是大约10％需要血液透析或移植用于终末期肾病的患者的潜在病理学。 在ADPKD，polycystin-1和polycystin-2中突变的两种蛋白质形成位于初级纤毛和质膜上的复合物，以促进细胞中钙离子的释放。 目前还没有食品和药物管理局（FDA）批准的治疗方法来治愈或减缓疾病的进展。 啮齿动物ADPKD模型并不完全模仿人类疾病，因此临床前结果并不总能成功转化为临床。 此外，许多这些潜在疗法的毒性导致患者退出临床试验。 结果：在这里，我们回顾了用于治疗ADPKD的临床试验中的化合物，我们研究了使用肾靶向方法的可行性，有可能拓宽治疗窗口，降低治疗相关毒性并提高已证明活性的药物的功效 在动物模型中。 我们提出了将肾靶向治疗与目前治疗方案相结合的建议，以实现治疗ADPKD的综合方法。 结论：许多化合物目前正在进行ADPKD的临床试验，但到目前为止，还没有一种化合物被FDA批准用于治疗这种疾病。 患者可以从有效的药物治疗中受益，特别是如果它可以针对肾脏，并且集中精力继续关注这一目标。

关键词: 常染色体显性多囊肾病（ADPKD），肾脏特异性治疗，肾脏疾病，靶向治疗，多囊肾病，常染色体疾病。

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