Title:Perspectives of Gene Therapies in Autosomal Dominant Polycystic Kidney Disease
VOLUME: 17 ISSUE: 1
Author(s):Yuchen Xu, Ao Li, Guanqing Wu* and Chaozhao Liang*
Affiliation:Institute/Department of Urology, The Anhui Province PKD Center, The First Affiliated Hospital of Anhui Medical University, Hefei, Anhui Province, 230022, Institute/Department of Urology, The Anhui Province PKD Center, The First Affiliated Hospital of Anhui Medical University, Hefei, Anhui Province, 230022, Anhui Province PKD Center, Institute/Department of Urology, The First Affiliated Hospital, Anhui Medical University, Hefei, 230022, Institute/Department of Urology, The Anhui Province PKD Center, The First Affiliated Hospital of Anhui Medical University, Hefei, Anhui Province, 230022
Keywords:Aberrant signaling pathways, ADPKD, causative genes, gene therapy, kidney disease, translational medicine.
Abstract: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.
Method: Although causative genes and aberrant signaling pathways have been investigated for decades,
lack of effective and less side-effect treatment for the disease still perplex vast clinicians. Therefore,
development of new therapeutic approaches for ADPKD is currently very much desired.
Conclusion: This review will center on pathogenesis of ADPKD, and thereafter gene transfer will be
discussed as potential treatment for the disease. New therapeutic interventions will bring further hope
to improve prognosis of this incurable disease.
