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Letter
Nature Medicine  9, 1323 - 1326 (2003)
Published online: 21 September 2003; | doi:10.1038/nm935

Inhibition of renal cystic disease development and progression by a vasopressin V2 receptor antagonist

Vincent H Gattone II1, Xiaofang Wang2, Peter C Harris2 & Vicente E Torres2

1  Anatomy and Cell Biology, Indiana University School of Medicine, 635 Barnhill Drive, Indianapolis, Indiana 46202, USA.

2  Division of Nephrology, Mayo Clinic, 200 First Street SW, Rochester, Minnesota 55905, USA.

Correspondence should be addressed to Vincent H Gattone II gattone@anatomy.iupui.edu or Vicente E Torres torres.vicente@mayo.edu
The polycystic kidney diseases (PKDs) are a group of genetic disorders causing significant renal failure and death in children and adults. There are no effective treatments. Two childhood forms, autosomal recessive PKD (ARPKD) and nephronophthisis (NPH), are characterized by collecting-duct cysts1, 2. We used animal models orthologous to the human disorders to test whether a vasopressin V2 receptor (VPV2R) antagonist, OPC31260, would be effective against early or established disease. Adenosine-3',5'-cyclic monophosphate (cAMP) has a major role in cystogenesis3, 4, and the VPV2R is the major cAMP agonist in the collecting duct5, 6. OPC31260 administration lowered renal cAMP, inhibited disease development and either halted progression or caused regression of established disease. These results indicate that OPC31260 may be an effective treatment for these disorders and that clinical trials should be considered.


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REFERENCE
Polycystic Disease of the Kidney
Nature Encyclopaedia of Life Sciences

REVIEWS
Autosomal dominant polycystic kidney disease (ADPKD, MIM 173900, PKD1 and PKD2 genes, protein products known as polycystin-1 and polycystin-2)
European Journal of Human Genetics Reviews (01 May 2004)
 See all 3 matches for Reviews

NEWS AND VIEWS
Another cystic mystery solved
Nature Genetics News and Views (01 Mar 2002)
The ins and outs of polycystin-2 as a calcium release channel
Nature Cell Biology News and Views (01 Mar 2002)
 See all 4 matches for News And Views

RESEARCH
Effective treatment of an orthologous model of autosomal dominant polycystic kidney disease
Nature Medicine Brief Communications (01 Apr 2004)
 See all 7 matches for Research

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Nature Medicine
ISSN: 1078-8956
EISSN: 1546-170X
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