常染色体显性多囊性肾病的基因治疗方法

Title:Perspectives of Gene Therapies in Autosomal Dominant Polycystic Kidney Disease

Volume: 17 Issue: 1

关键词: 异常信号通路，常染色体显性多囊性肾病，致病基因，基因治疗，肾病，转译医学

摘要: Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease in the clinic. The predominant clinical manifestation is bilateral and progressive cysts formation in the kidneys, impairs normal renal parenchyma, and ultimately leads to endstage renal disease (ESRD). ADPKD is a heterogenic disease which is resulted from the mutations of PKD1 or PKD2 genes which encode polycystin-1 (PC1) and -2 (PC2), thereby multiple cell signaling pathways are involved.

Cite this article as:

Perspectives of Gene Therapies in Autosomal Dominant Polycystic Kidney Disease, Current Gene Therapy 2017; 17 (1) . https://dx.doi.org/10.2174/1566523217666170510152808