Novel targets for the treatment of autosomal dominant polycystic kidney disease
- 8 February 2010
- journal article
- review article
- Published by Informa UK Limited in Expert Opinion on Investigational Drugs
- Vol. 19 (3), 315-328
- https://doi.org/10.1517/13543781003588491
Abstract
Importance of the field: Autosomal dominant (AD) polycystic kidney disease (PKD) is the most common life-threatening hereditary disorder. There is currently no therapy that slows or prevents cyst formation and kidney enlargement in humans. An increasing number of animal studies have advanced our understanding of molecular and cellular targets of PKD. Areas covered in the review: The purpose of this review is to summarize the molecular and cellular targets involved in cystogenesis and to update on the promising therapies that are being developed and tested based on knowledge of these molecular and cellular targets. What the reader will gain: Insight into the pathogenesis of PKD and how a better understanding of the pathogenesis of PKD has led to the development of potential therapies to inhibit cyst formation and/or growth and improve kidney function. Take home message: The results of animal studies in PKD have led to the development of clinical trials testing potential new therapies to reduce cyst formation and/or growth. A vasopressin V2 receptor antagonist, mTOR inhibitors, blockade of the renin–angiotensin system and statins that reduce cyst formation and improve renal function in animal models of PKD are being tested in interventional studies in humans.Keywords
This publication has 104 references indexed in Scilit:
- Autosomal dominant polycystic kidney disease: the last 3 yearsKidney International, 2009
- Cyst Formation in Kidney via B-Raf Signaling in the PKD2 Transgenic MicePublished by Elsevier BV ,2009
- A tumor necrosis factor-α–mediated pathway promoting autosomal dominant polycystic kidney diseaseNature Medicine, 2008
- Nek8 Regulates the Expression and Localization of Polycystin-1 and Polycystin-2Journal of the American Society of Nephrology, 2008
- Cyst formation and activation of the extracellular regulated kinase pathway after kidney specific inactivation of Pkd1Human Molecular Genetics, 2008
- Sirolimus for Angiomyolipoma in Tuberous Sclerosis Complex or LymphangioleiomyomatosisNew England Journal of Medicine, 2008
- Disruption of Intraflagellar Transport in Adult Mice Leads to Obesity and Slow-Onset Cystic Kidney DiseaseCurrent Biology, 2007
- Hyperproliferation of PKD1 cystic cells is induced by insulin-like growth factor-1 activation of the Ras/Raf signalling systemKidney International, 2007
- Triptolide is a traditional Chinese medicine-derived inhibitor of polycystic kidney diseaseProceedings of the National Academy of Sciences, 2007
- Rheb is a direct target of the tuberous sclerosis tumour suppressor proteinsNature, 2003