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.Keywords: Aberrant signaling pathways, ADPKD, causative genes, gene therapy, kidney disease, translational medicine.
Current Gene Therapy
Title:Perspectives of Gene Therapies in Autosomal Dominant Polycystic Kidney Disease
Volume: 17 Issue: 1
Author(s): Yuchen Xu, Ao Li, Guanqing Wu*Chaozhao Liang*
Affiliation:
- Anhui Province PKD Center, Institute/Department of Urology, The First Affiliated Hospital, Anhui Medical University, Hefei, 230022,China
- Institute/Department of Urology, The Anhui Province PKD Center, The First Affiliated Hospital of Anhui Medical University, Hefei, Anhui Province, 230022,China
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.Export Options
About this article
Cite this article as:
Xu Yuchen , Li Ao, Wu Guanqing*, Liang Chaozhao*, Perspectives of Gene Therapies in Autosomal Dominant Polycystic Kidney Disease, Current Gene Therapy 2017; 17 (1) . https://dx.doi.org/10.2174/1566523217666170510152808
DOI https://dx.doi.org/10.2174/1566523217666170510152808 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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