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Targeting latent TGFβ release in muscular dystrophy.
Ceco E, Bogdanovich S, Gardner B, Miller T, DeJesus A, Earley JU, Hadhazy M, Smith LR, Barton ER, Molkentin JD, McNally EM. Ceco E, et al. Among authors: barton er. Sci Transl Med. 2014 Oct 22;6(259):259ra144. doi: 10.1126/scitranslmed.3010018. Sci Transl Med. 2014. PMID: 25338755 Free PMC article.
PTC124 targets genetic disorders caused by nonsense mutations.
Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL. Welch EM, et al. Among authors: barton er. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. Nature. 2007. PMID: 17450125
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