Novel therapeutic approaches to autosomal dominant polycystic kidney disease.
Novel therapeutic approaches to autosomal dominant polycystic kidney disease.
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DOI：
10.1016/j.trsl.2014.11.003
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发表时间：
2015-04
期刊：
影响因子：
--
通讯作者：
Torres VE
中科院分区：
文献类型：
--
作者：
LaRiviere WB;Irazabal MV;Torres VE
常染色体显性遗传性多囊肾病（ADPKD）是一种遗传性疾病，其特征是肾囊肿的进行性生长，随着时间的推移，破坏肾实质的结构，通常在60岁时导致肾衰竭。ADPKD很常见，是全球肾衰竭的主要原因。目前，没有FDA批准的治疗方法，现有的护理标准主要是支持性的。然而，对该疾病的分子生物学理解的重大进展激发了对潜在新疗法的研究。几种旨在减缓或阻止ADPKD进展的药物已在临床前模型和临床试验中显示出希望，包括加压素受体拮抗剂和生长抑素类似物。这篇文章检查了ADPKD分子治疗的基本原理的文献，并回顾了其适应症为人类患者的疾病的现有临床证据。
Autosomal dominant polycystic kidney disease (ADPKD) is an inherited disorder characterized by the progressive growth of renal cysts that, over time, destroy the architecture of the renal parenchyma and typically lead to kidney failure by the 6th decade of life. ADPKD is common and represents a leading cause of renal failure worldwide. Currently, there are no FDA approved treatment for the disease, and the existing standard of care is primarily supportive in nature. However, significant advances in the understanding of the molecular biology of the disease have inspired investigation into potential new therapies. Several drugs designed to slow or arrest the progression of ADPKD have shown promise in pre-clinical models and clinical trials, including vasopressin receptor antagonists and somatostatin analogs. This article examines literature underlying the rationale for molecular therapies for ADPKD and reviews the existing clinical evidence for their indication for human patients with the disease.
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