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Gene therapy for inborn errors of liver metabolism.
Brunetti-Pierri N, Lee B. Brunetti-Pierri N, et al. Mol Genet Metab. 2005 Sep-Oct;86(1-2):13-24. doi: 10.1016/j.ymgme.2005.07.015. Mol Genet Metab. 2005. PMID: 16154372 Review.
Discovery of drug mode of action and drug repositioning from transcriptional responses.
Iorio F, Bosotti R, Scacheri E, Belcastro V, Mithbaokar P, Ferriero R, Murino L, Tagliaferri R, Brunetti-Pierri N, Isacchi A, di Bernardo D. Iorio F, et al. Proc Natl Acad Sci U S A. 2010 Aug 17;107(33):14621-6. doi: 10.1073/pnas.1000138107. Epub 2010 Aug 2. Proc Natl Acad Sci U S A. 2010. PMID: 20679242 Free PMC article.
Phenylbutyrate therapy for maple syrup urine disease.
Brunetti-Pierri N, Lanpher B, Erez A, Ananieva EA, Islam M, Marini JC, Sun Q, Yu C, Hegde M, Li J, Wynn RM, Chuang DT, Hutson S, Lee B. Brunetti-Pierri N, et al. Hum Mol Genet. 2011 Feb 15;20(4):631-40. doi: 10.1093/hmg/ddq507. Epub 2010 Nov 23. Hum Mol Genet. 2011. PMID: 21098507 Free PMC article. Clinical Trial.
275 results