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Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C mice.
Nietupski JB, Pacheco JJ, Chuang WL, Maratea K, Li L, Foley J, Ashe KM, Cooper CG, Aerts JM, Copeland DP, Scheule RK, Cheng SH, Marshall J. Nietupski JB, et al. Among authors: scheule rk. Mol Genet Metab. 2012 Apr;105(4):621-8. doi: 10.1016/j.ymgme.2012.01.020. Epub 2012 Feb 1. Mol Genet Metab. 2012. PMID: 22366055
Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy.
Marshall J, McEachern KA, Chuang WL, Hutto E, Siegel CS, Shayman JA, Grabowski GA, Scheule RK, Copeland DP, Cheng SH. Marshall J, et al. Among authors: scheule rk. J Inherit Metab Dis. 2010 Jun;33(3):281-9. doi: 10.1007/s10545-010-9072-z. Epub 2010 Mar 25. J Inherit Metab Dis. 2010. PMID: 20336375 Free PMC article.
Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease.
Ashe KM, Bangari D, Li L, Cabrera-Salazar MA, Bercury SD, Nietupski JB, Cooper CG, Aerts JM, Lee ER, Copeland DP, Cheng SH, Scheule RK, Marshall J. Ashe KM, et al. Among authors: scheule rk. PLoS One. 2011;6(6):e21758. doi: 10.1371/journal.pone.0021758. Epub 2011 Jun 29. PLoS One. 2011. PMID: 21738789 Free PMC article.
Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy.
Hurlbut GD, Ziegler RJ, Nietupski JB, Foley JW, Woodworth LA, Meyers E, Bercury SD, Pande NN, Souza DW, Bree MP, Lukason MJ, Marshall J, Cheng SH, Scheule RK. Hurlbut GD, et al. Among authors: scheule rk. Mol Ther. 2010 Nov;18(11):1983-94. doi: 10.1038/mt.2010.175. Epub 2010 Aug 24. Mol Ther. 2010. PMID: 20736932 Free PMC article.
Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease.
Cabrera-Salazar MA, Deriso M, Bercury SD, Li L, Lydon JT, Weber W, Pande N, Cromwell MA, Copeland D, Leonard J, Cheng SH, Scheule RK. Cabrera-Salazar MA, et al. Among authors: scheule rk. PLoS One. 2012;7(8):e43310. doi: 10.1371/journal.pone.0043310. Epub 2012 Aug 17. PLoS One. 2012. PMID: 22912851 Free PMC article.
109 results