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Sci. Signal., 13 July 2010
Vol. 3, Issue 130, p. ec211
[DOI: 10.1126/scisignal.3130ec211]


Medicine Correcting a Liver Problem

Stella M. Hurtley

Science, AAAS, Cambridge CB2 1LQ, UK

The classical form of {alpha}1-antitrypsin (AT) deficiency is caused by a point mutation that alters the folding and causes intracellular aggregation of AT—an abundant liver-derived plasma glycoprotein. AT deficiency is the most common genetic cause of liver disease in childhood and can also lead to cirrhosis and/or hepatocellular carcinoma in adulthood. Carbamazepine is a drug known to be well tolerated in humans that enhances the intracellular degradation process known as autophagy. Now, Hidvegi et al. (see the Perspective by Sifers) show that carbamazepine can reduce the severity of liver disease in a mouse model of AT deficiency by enhancing the degradation of misfolded accumulated AT.

T. Hidvegi, M. Ewing, P. Hale, C. Dippold, C. Beckett, C. Kemp, N. Maurice, A. Mukherjee, C. Goldbach, S. Watkins, G. Michalopoulos, D. H. Perlmutter, An autophagy-enhancing drug promotes degradation of mutant {alpha}1-antitrypsin Z and reduces hepatic fibrosis. Science 329, 229–232 (2010). [Abstract] [Full Text]

R. N. Sifers, Clearing conformational disease. Science 329, 154–155 (2010). [Summary] [Full Text]

Citation: S. M. Hurtley, Correcting a Liver Problem. Sci. Signal. 3, ec211 (2010).

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