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中华肾病研究电子杂志 ›› 2017, Vol. 06 ›› Issue (01) : 34 -38. doi: 10.3877/cma.j.issn.2095-3216.2017.01.008

所属专题: 文献

综述

常染色体显性遗传性多囊肾病发病机制及治疗靶点的研究进展
陈熳1, 谢院生2,()   
  1. 1. 100853 北京,解放军总医院肾脏病科、解放军肾脏病研究所、肾脏疾病国家重点实验室、国家慢性肾病临床医学研究中心;300071 天津,南开大学医学院
    2. 100853 北京,解放军总医院肾脏病科、解放军肾脏病研究所、肾脏疾病国家重点实验室、国家慢性肾病临床医学研究中心
  • 收稿日期:2016-12-23 出版日期:2017-02-28
  • 通信作者: 谢院生
  • 基金资助:
    国家自然科学基金项目(81473531); 国家重点研发计划(2016YFC0901502)

Progress of research on pathogenesis and therapeutic targets of autosomal dominant polycystic kidney disease

Man Chen1, Yuansheng Xie2,()   

  1. 1. Department of Nephrology, Chinese PLA General Hospital, Chinese PLA Institute of Nephrology, State Key Laboratory of Kidney Diseases, National Clinical Research Center for Kidney Diseases, Beijing 100853; Nankai University Medical College, Tianjin 300071, China
    2. Department of Nephrology, Chinese PLA General Hospital, Chinese PLA Institute of Nephrology, State Key Laboratory of Kidney Diseases, National Clinical Research Center for Kidney Diseases, Beijing 100853
  • Received:2016-12-23 Published:2017-02-28
  • Corresponding author: Yuansheng Xie
  • About author:
    Corresponding author: Xie Yuansheng, Email:
引用本文:

陈熳, 谢院生. 常染色体显性遗传性多囊肾病发病机制及治疗靶点的研究进展[J]. 中华肾病研究电子杂志, 2017, 06(01): 34-38.

Man Chen, Yuansheng Xie. Progress of research on pathogenesis and therapeutic targets of autosomal dominant polycystic kidney disease[J]. Chinese Journal of Kidney Disease Investigation(Electronic Edition), 2017, 06(01): 34-38.

常染色体显性多囊肾病(ADPKD)是一种常见的遗传性肾脏病,以双肾多发液性囊肿为特征。ADPKD患病率约1/1 000,是尿毒症的第四位病因。大约有50%的多囊肾患者在60岁以前会进入终末期肾病阶段,缺乏特效的治疗措施。近年来,ADPKD分子机制研究的进展,为寻找其治疗靶点提供了新方向,针对不同靶点的药物在动物实验和临床试验中取得了一定的效果。本文将对ADPKD的发病机制及药物研究进展做一综述。

Autosomal dominant polycystic kidney disease (ADPKD) is a common inherited kidney disease, which is characterized by bilateral renal multiple fluid-filled cysts. ADPKD is the fourth cause of uremia, with a prevalence of about 1/1 000. Approximately half of the patients with ADPKD will progress to end-stage renal disease before the age of 60 years, but there has been not any very effective therapies. In recent years, advances in the molecular mechanisms of this disease have provided new directions for seeking the therapeutic targets. Preclinical models and clinical trials of the novel therapies have achieved positive results. This review focused on the progress of studies on pathogenesis and therapies of ADPKD.

图1 针对常染色体显性遗传性多囊肾病发病机制的治疗靶点与药物
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