常染色体显性多囊肾病的分子靶向药物治疗研究进展

Research Progress of Molecular Targeted Drugs in Autosomal Dominant Polycystic Kidney Disease

常染色体显性多囊肾病(ADPKD)是一种单基因遗传性肾脏疾病,其特征是囊肿进行性形成,导致肾脏体积增加和肾功能下降,最后进展为终末期肾病。既往临床上对ADPKD的治疗以对症支持治疗为主,无法延缓多囊肾病的病情进展,临床上亟需针对ADPKD的有效治疗方案。近年来,随着人们对ADPKD病理生理机制研究的不断深入,针对ADPKD致病靶点和相关信号通路的分子靶向药物研发不断取得进展突破,目前已有多种ADPKD的分子靶向药物进入临床试验阶段。本文就ADPKD分子靶向药物研究进展进行综述,以期对该病治疗及新药研发有所裨益。

Autosomal dominant polycystic kidney disease(ADPKD)is a single-gene inherited kidney disease characterized by the progressive formation of cysts,leading to increased kidney volume and decreased kidney function,and resulting in end-stage renal disease.The previous clinical treatment of ADPKD is mainly symptomatic supportive treatment,which cannot delay the progression of polycystic kidney disease.Therefore,an effective treatment plan for ADPKD is urgently needed clinically.In recent years,with the continuous in-depth research on the pathophysiological mechanism of ADPKD,the research and development of molecular targeting drugs for the pathogenic targets of ADPKD and related signaling pathways have made continuous breakthroughs.Currently,a variety of molecular targeting drugs for ADPKD have entered the stage of clinical trials.In this paper,the research progress of molecular targeting drugs of ADPKD was reviewed,in order to be helpful for the treatment of ADPKD and the development of new drugs.