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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. Among authors: xu x. 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.
Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease.
Ottinger EA, Kao ML, Carrillo-Carrasco N, Yanjanin N, Shankar RK, Janssen M, Brewster M, Scott I, Xu X, Cradock J, Terse P, Dehdashti SJ, Marugan J, Zheng W, Portilla L, Hubbs A, Pavan WJ, Heiss J, Vite CH, Walkley SU, Ory DS, Silber SA, Porter FD, Austin CP, McKew JC. Ottinger EA, et al. Among authors: xu x. Curr Top Med Chem. 2014;14(3):330-9. doi: 10.2174/1568026613666131127160118. Curr Top Med Chem. 2014. PMID: 24283970 Free PMC article. Review.
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial.
Ory DS, Ottinger EA, Farhat NY, King KA, Jiang X, Weissfeld L, Berry-Kravis E, Davidson CD, Bianconi S, Keener LA, Rao R, Soldatos A, Sidhu R, Walters KA, Xu X, Thurm A, Solomon B, Pavan WJ, Machielse BN, Kao M, Silber SA, McKew JC, Brewer CC, Vite CH, Walkley SU, Austin CP, Porter FD. Ory DS, et al. Among authors: xu x. Lancet. 2017 Oct 14;390(10104):1758-1768. doi: 10.1016/S0140-6736(17)31465-4. Epub 2017 Aug 10. Lancet. 2017. PMID: 28803710 Free PMC article. Clinical Trial.
A target-agnostic screen identifies approved drugs to stabilize the endoplasmic reticulum-resident proteome.
Henderson MJ, Trychta KA, Yang SM, Bäck S, Yasgar A, Wires ES, Danchik C, Yan X, Yano H, Shi L, Wu KJ, Wang AQ, Tao D, Zahoránszky-Kőhalmi G, Hu X, Xu X, Maloney D, Zakharov AV, Rai G, Urano F, Airavaara M, Gavrilova O, Jadhav A, Wang Y, Simeonov A, Harvey BK. Henderson MJ, et al. Among authors: xu x. Cell Rep. 2021 Apr 27;35(4):109040. doi: 10.1016/j.celrep.2021.109040. Cell Rep. 2021. PMID: 33910017 Free PMC article.
Activation of mitochondrial TRAP1 stimulates mitochondria-lysosome crosstalk and correction of lysosomal dysfunction.
Chen FW, Davies JP, Calvo R, Chaudhari J, Dolios G, Taylor MK, Patnaik S, Dehdashti J, Mull R, Dranchack P, Wang A, Xu X, Hughes E, Southall N, Ferrer M, Wang R, Marugan JJ, Ioannou YA. Chen FW, et al. Among authors: xu x. iScience. 2022 Aug 14;25(9):104941. doi: 10.1016/j.isci.2022.104941. eCollection 2022 Sep 16. iScience. 2022. PMID: 36065186 Free PMC article.
Benserazide racemate and enantiomers induce fetal globin gene expression in vivo: Studies to guide clinical development for beta thalassemia and sickle cell disease.
Pace BS, Perrine S, Li B, Makala L, Xu H, Takezaki M, Wolf RF, Wang A, Xu X, Huang J, Alimardanov A, Tawa GJ, Sangerman J, Faller A, Zheng W, Toney L, Haugabook SJ. Pace BS, et al. Among authors: xu h, xu x. Blood Cells Mol Dis. 2021 Jul;89:102561. doi: 10.1016/j.bcmd.2021.102561. Epub 2021 Mar 12. Blood Cells Mol Dis. 2021. PMID: 33744514 Free PMC article.
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