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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: cheng sh. 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: cheng sh. Hum Gene Ther. 2008 Jun;19(6):609-21. doi: 10.1089/hum.2008.010. Hum Gene Ther. 2008. PMID: 18500944
Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease.
Zhu Y, Jiang JL, Gumlaw NK, Zhang J, Bercury SD, Ziegler RJ, Lee K, Kudo M, Canfield WM, Edmunds T, Jiang C, Mattaliano RJ, Cheng SH. Zhu Y, et al. Among authors: cheng sh. Mol Ther. 2009 Jun;17(6):954-63. doi: 10.1038/mt.2009.37. Epub 2009 Mar 10. Mol Ther. 2009. PMID: 19277015 Free PMC article.
1,012 results