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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: aerts jm. PLoS One. 2011;6(6):e21758. doi: 10.1371/journal.pone.0021758. Epub 2011 Jun 29. PLoS One. 2011. PMID: 21738789 Free PMC article.
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: aerts jm. 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
Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease.
Liefhebber JMP, Brasser G, Spronck EA, Ottenhoff R, Paerels L, Ferraz MJ, Schwarz LK, Efthymiopoulou N, Kuo CL, Montenegro-Miranda PS, Evers MM, Aerts JMFG, Liu YP. Liefhebber JMP, et al. Among authors: aerts jmfg. Mol Ther Methods Clin Dev. 2024 Nov 12;32(4):101375. doi: 10.1016/j.omtm.2024.101375. eCollection 2024 Dec 12. Mol Ther Methods Clin Dev. 2024. PMID: 39687734 Free PMC article.
461 results