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Endocrine/metabolic disease


ATP-binding cassette sub-family G WHITE member 2 (ABCG2; MXR; BCRP1)

In vitro studies identified small molecule structural correctors of ABCG2 that could help treat gout. The Q141K mutation in ABCG2 decreases urate transport and has been associated with an increased risk of developing gout. SAR studies identified a small molecule that corrected the folding of the Q141K mutant ABCG2 and increased expression of the protein in cultured cells compared with vehicle. Next steps include screening for and developing additional Q141K corrector molecules to treat gout.

SciBX 6(12); doi:10.1038/scibx.2013.290
Published online March 28, 2013

Patent application filed; available for licensing

Woodward, O.M. et al. Proc. Natl. Acad. Sci. USA; published online
March 14, 2013;
Contact: William B. Guggino, The Johns Hopkins University School of Medicine, Baltimore, Md.
Contact: Owen M. Woodward, same affiliation as above
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