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ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice.
Abera MB, Xiao J, Nofziger J, Titus S, Southall N, Zheng W, Moritz KE, Ferrer M, Cherry JJ, Androphy EJ, Wang A, Xu X, Austin C, Fischbeck KH, Marugan JJ, Burnett BG. Abera MB, et al. Among authors: burnett bg. JCI Insight. 2016 Nov 17;1(19):e88427. doi: 10.1172/jci.insight.88427. JCI Insight. 2016. PMID: 27882347 Free PMC article.
Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy.
Rietz A, Li H, Quist KM, Cherry JJ, Lorson CL, Burnett BG, Kern NL, Calder AN, Fritsche M, Lusic H, Boaler PJ, Choi S, Xing X, Glicksman MA, Cuny GD, Androphy EJ, Hodgetts KJ. Rietz A, et al. Among authors: burnett bg. J Med Chem. 2017 Jun 8;60(11):4594-4610. doi: 10.1021/acs.jmedchem.6b01885. Epub 2017 May 19. J Med Chem. 2017. PMID: 28481536 Free PMC article.
A combinatorial approach increases SMN level in SMA model mice.
Dumas SA, Villalón E, Bergman EM, Wilson KJ, Marugan JJ, Lorson CL, Burnett BG. Dumas SA, et al. Among authors: burnett bg. Hum Mol Genet. 2022 Aug 25;31(17):2989-3000. doi: 10.1093/hmg/ddac068. Hum Mol Genet. 2022. PMID: 35419606 Free PMC article.
Mitochondrial abnormalities in spinal and bulbar muscular atrophy.
Ranganathan S, Harmison GG, Meyertholen K, Pennuto M, Burnett BG, Fischbeck KH. Ranganathan S, et al. Among authors: burnett bg. Hum Mol Genet. 2009 Jan 1;18(1):27-42. doi: 10.1093/hmg/ddn310. Epub 2008 Sep 29. Hum Mol Genet. 2009. PMID: 18824496 Free PMC article.
Regulation of SMN protein stability.
Burnett BG, Muñoz E, Tandon A, Kwon DY, Sumner CJ, Fischbeck KH. Burnett BG, et al. Mol Cell Biol. 2009 Mar;29(5):1107-15. doi: 10.1128/MCB.01262-08. Epub 2008 Dec 22. Mol Cell Biol. 2009. PMID: 19103745 Free PMC article.
41 results