2019
DOI: 10.3233/trd-190040
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Novel Treatments for Polycystic Kidney Disease

Abstract: Polycystic kidney disease (PKD) and nephronophthisis are common manifestation of ciliopathies. PKD is the most common genetic renal condition; it affects 12.5 million people worldwide. PKD is a great example of decades of translational research leading to the discovery of novel treatments and significant number of clinical trials. This review will concentrate on the basic molecular and cellular pathophysiology that led to the development of therapeutic targets for PKD.

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Cited by 2 publications
(3 citation statements)
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“…There are multiple pathways and signaling molecules that cause phenotypic changes in renal epithelial cells, which ultimately lead to cyst formation. How malfunctioning PKD proteins affect these pathways is not yet fully understood [ 6 ]. For the implementation of novel treatment targets and strategies to preserve renal function in patients suffering from ADPKD, it is crucial to identify the cellular mechanisms and understand the pathophysiological cascade involved in the formation and growth of renal cysts in ADPKD.…”
Section: Introductionmentioning
confidence: 99%
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“…There are multiple pathways and signaling molecules that cause phenotypic changes in renal epithelial cells, which ultimately lead to cyst formation. How malfunctioning PKD proteins affect these pathways is not yet fully understood [ 6 ]. For the implementation of novel treatment targets and strategies to preserve renal function in patients suffering from ADPKD, it is crucial to identify the cellular mechanisms and understand the pathophysiological cascade involved in the formation and growth of renal cysts in ADPKD.…”
Section: Introductionmentioning
confidence: 99%
“…Furthermore, there is a broad consensus regarding the important roles that fluid secretion and cell proliferation play in the multidimensional cyst development and enlargement process [ 6 , 9 ]. Enhanced fluid secretion is supposed to be mediated by the cystic fibrosis transmembrane conductance regulator (CFTR), which facilitates cAMP-stimulated chloride secretion in ADPKD cysts [ 10 , 11 ].…”
Section: Introductionmentioning
confidence: 99%
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