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A New Therapeutic Strategy for Autosomal Dominant Polycystic Kidney Disease: Activation of AMP Kinase by Metformin

Michael J. Caplan

2011

Abstract

Abstract : Autosomal dominant polycystic kidney disease is a common inherited disorder. Patients are born with normal kidneys but, over the course of decades, they develop large fluid filled cysts that damage the normal kidney tissue. The damage caused by these cysts can lead ultimately to kidney failure, necessitating kidney transplantation or dialysis. There are currently no approved medications for this condition. Recent research reveals that cyst formation is due in part both to inappropriate cell growth and fluid secretion. The enzyme AMPK controls a number of cellular pathways, including those involved in cell growth and fluid secretion. Drugs that activate AMPK, therefore, may constitute an effective therapeutic option for slowing or preventing cyst growth. This research project is aimed at examining the potential of an approved, widely used, inexpensive and low-toxicity drug that can activate AMPK as a potential therapy for the treatment of polycystic kidney disease.

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