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Structure-dependent impairment of intracellular apolipoprotein E4 trafficking and its detrimental effects are rescued by small-molecule structure correctors.
Brodbeck J, McGuire J, Liu Z, Meyer-Franke A, Balestra ME, Jeong DE, Pleiss M, McComas C, Hess F, Witter D, Peterson S, Childers M, Goulet M, Liverton N, Hargreaves R, Freedman S, Weisgraber KH, Mahley RW, Huang Y. Brodbeck J, et al. Among authors: balestra me. J Biol Chem. 2011 May 13;286(19):17217-26. doi: 10.1074/jbc.M110.217380. Epub 2011 Mar 18. J Biol Chem. 2011. PMID: 21454574 Free PMC article.
Small molecule structure correctors abolish detrimental effects of apolipoprotein E4 in cultured neurons.
Chen HK, Liu Z, Meyer-Franke A, Brodbeck J, Miranda RD, McGuire JG, Pleiss MA, Ji ZS, Balestra ME, Walker DW, Xu Q, Jeong DE, Budamagunta MS, Voss JC, Freedman SB, Weisgraber KH, Huang Y, Mahley RW. Chen HK, et al. Among authors: balestra me. J Biol Chem. 2012 Feb 17;287(8):5253-66. doi: 10.1074/jbc.M111.276162. Epub 2011 Dec 12. J Biol Chem. 2012. PMID: 22158868 Free PMC article.
Genetic correction of tauopathy phenotypes in neurons derived from human induced pluripotent stem cells.
Fong H, Wang C, Knoferle J, Walker D, Balestra ME, Tong LM, Leung L, Ring KL, Seeley WW, Karydas A, Kshirsagar MA, Boxer AL, Kosik KS, Miller BL, Huang Y. Fong H, et al. Among authors: balestra me. Stem Cell Reports. 2013 Aug 29;1(3):226-34. doi: 10.1016/j.stemcr.2013.08.001. eCollection 2013. Stem Cell Reports. 2013. PMID: 24319659 Free PMC article.
Rare variants in the neuronal ceroid lipofuscinosis gene MFSD8 are candidate risk factors for frontotemporal dementia.
Geier EG, Bourdenx M, Storm NJ, Cochran JN, Sirkis DW, Hwang JH, Bonham LW, Ramos EM, Diaz A, Van Berlo V, Dokuru D, Nana AL, Karydas A, Balestra ME, Huang Y, Russo SP, Spina S, Grinberg LT, Seeley WW, Myers RM, Miller BL, Coppola G, Lee SE, Cuervo AM, Yokoyama JS. Geier EG, et al. Among authors: balestra me. Acta Neuropathol. 2019 Jan;137(1):71-88. doi: 10.1007/s00401-018-1925-9. Epub 2018 Oct 31. Acta Neuropathol. 2019. PMID: 30382371 Free PMC article.
23 results