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Endocrine/metabolic disease

Endocrine/metabolic disease

Branched chain ketoacid dehydrogenase kinase (BCKDK; BDK)

In vitro and mouse studies identified a BDK inhibitor that could help treat diseases associated with accumulation of branched-chain amino acids (BCAAs), including the
genetic disorder branched-chain ketoaciduria, obesity
and diabetes. Crystal structures of BDK in complex
with known inhibitors led to the rational design of
(S)-a-chloro-phenylpropionic acid, which inhibits the kinase at an allosteric site. In wild-type mice, injection of the compound decreased both plasma BCAA levels and BDK activity in multiple tissue types compared with vehicle injection. Next steps could include testing the compound in disease models.

SciBX 6(24); doi:10.1038/scibx.2013.603
Published online June 20, 2013

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Tso, S.-C. et al. Proc. Natl. Acad. Sci. USA; published online May 28, 2013;
Contact: David T. Chuang, The University of Texas Southwestern Medical Center, Dallas, Texas
Contact: Uttam K. Tambar, same affiliation as above