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Page 1
Design, Synthesis, and Evaluation of a Luminescent Cholesterol Mimic.
Work EM, Ferraudi G, Kiefer L, Liu G, Grigalunas M, Bhardwaj A, Kaur R, Dempsey JM, Wüstner D, Helquist P, Wiest O. Work EM, et al. J Org Chem. 2021 Jan 15;86(2):1612-1621. doi: 10.1021/acs.joc.0c02460. Epub 2020 Dec 28. J Org Chem. 2021. PMID: 33369429 Free PMC article.
Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance of Cholesterol Accumulation in Niemann-Pick C1 Fibroblasts.
Cruz DL, Pipalia N, Mao S, Gadi D, Liu G, Grigalunas M, O'Neill M, Quinn TR, Kipper A, Ekebergh A, Dimmling A, Gartner C, Melancon BJ, Wagner FF, Holson E, Helquist P, Wiest O, Maxfield FR. Cruz DL, et al. ACS Pharmacol Transl Sci. 2021 May 27;4(3):1136-1148. doi: 10.1021/acsptsci.1c00033. eCollection 2021 Jun 11. ACS Pharmacol Transl Sci. 2021. PMID: 34151204 Free PMC article.
2-Hydroxypropyl-β-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 disease.
Davidson J, Molitor E, Moores S, Gale SE, Subramanian K, Jiang X, Sidhu R, Kell P, Zhang J, Fujiwara H, Davidson C, Helquist P, Melancon BJ, Grigalunas M, Liu G, Salahi F, Wiest O, Xu X, Porter FD, Pipalia NH, Cruz DL, Holson EB, Schaffer JE, Walkley SU, Maxfield FR, Ory DS. Davidson J, et al. Biochim Biophys Acta Mol Cell Biol Lipids. 2019 Oct;1864(10):1545-1561. doi: 10.1016/j.bbalip.2019.04.011. Epub 2019 Apr 30. Biochim Biophys Acta Mol Cell Biol Lipids. 2019. PMID: 31051283 Free PMC article.
Normalization of Hepatic Homeostasis in the Npc1nmf164 Mouse Model of Niemann-Pick Type C Disease Treated with the Histone Deacetylase Inhibitor Vorinostat.
Munkacsi AB, Hammond N, Schneider RT, Senanayake DS, Higaki K, Lagutin K, Bloor SJ, Ory DS, Maue RA, Chen FW, Hernandez-Ono A, Dahlson N, Repa JJ, Ginsberg HN, Ioannou YA, Sturley SL. Munkacsi AB, et al. J Biol Chem. 2017 Mar 17;292(11):4395-4410. doi: 10.1074/jbc.M116.770578. Epub 2016 Dec 28. J Biol Chem. 2017. PMID: 28031458 Free PMC article.