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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
Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice.
Ziegler RJ, Bercury SD, Fidler J, Zhao MA, Foley J, Taksir TV, Ryan S, Hodges BL, Scheule RK, Shihabuddin LS, Cheng SH. Ziegler RJ, et al. Among authors: scheule rk. Hum Gene Ther. 2008 Jun;19(6):609-21. doi: 10.1089/hum.2008.010. Hum Gene Ther. 2008. PMID: 18500944
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.
109 results