Perspectives of Gene Therapies in Autosomal Dominant Polycystic Kidney Disease.

Perspectives of Gene Therapies in Autosomal Dominant Polycystic Kidney Disease.
Curr Gene Ther. 2017 May 10;:
Authors: Xu Y, Li A, Wu G, Liang C
Abstract
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 end-stage 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. 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. This review will center on pathogenesis of ADPKD, and thereafter gene transferring will be discussed as potential treatment for the disease. New therapeutic interventions will bring further hope to improve prognosis of this incurable disease.
PMID: 28494735 [PubMed - as supplied by publisher]

Source: Current Gene Therapy - May 10, 2017 Category: Genetics & Stem Cells Authors: Xu Y, Li A, Wu G, Liang C Tags: Curr Gene Ther Source Type: research