Chronic renal failure in a mouse model of human adenine phosphoribosyltransferase deficiency.. Stockelman, Michael G., John N. Lorenz, Frost N. Smith, Gregory P. Boivin, Amrik Sahota, Jay A. Tischfield, and Peter J. Stambrook. Department of Cell Biology, Neurobiology and Anatomy, University of Cincinnati, College of Medicine, Cincinnati, OH 45267-0521. Department of Molecular and Cellular Physiology, University of Cincinnati, College of Medicine, Cincinnati, OH 45267-0567. Department of Pathology and Laboratory Medicine, University of Cincinnati, College of Medicine, Cincinnati, OH 45267-0529. Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis, IN 46202-5251
APStracts 5:0059F, 1998.
In humans, adenine phosphoribosyltransferase (APRT; EC 2.4.2.7) deficiency can manifest as nephrolithiasis, interstitial nephritis and chronic renal failure. APRT catalyzes synthesis of AMP from adenine and 5-phosphoribosyl-1-pyrophosphate. In the absence of APRT, 2,8-dihydroxyadenine (DHA) is produced from adenine by xanthine dehydrogenase (XDH), and can precipitate in the renal interstitium, resulting in kidney disease. Treatment with allopurinol controls formation of DHA stones by inhibiting XDH activity. Kidney disease in APRT-deficient mice resembles that seen in humans. By age 12 weeks, APRT-deficient male mice are, on average, mildly anemic and smaller than normal males. They have extensive renal interstitial damage (assessed by image analysis), elevated blood urea nitrogen (BUN), and their creatinine clearance rates, measuring excretion of infused creatinine as an estimate of GFR, are about half that of wild type males. APRT-deficient males treated with allopurinol in the drinking water had normal BUN and less extensive visible renal damage, but creatinine clearance remained low. Throughout their lifespan, homozygous null female mice manifested significantly less renal damage than homozygous null males of the same age. APRT-deficient females showed no significant impairment of GFR at 12 weeks age. Consequences of APRT deficiency in male mice are more pronounced than in females, possibly due to differences in rates of adenine or DHA synthesis, or to sex-determined responses of the kidneys.

Received 25 August 1997; accepted in final form 27 February 1998.
APS Manuscript Number F275-7.
Article publication pending Am. J. Physiol. (Renal Physiology).
ISSN 1080-4757 Copyright 1998 The American Physiological Society.
Published in APStracts on 9 March 1998