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Gene therapy for the lysosomal storage disorders.
Cabrera-Salazar MA, Novelli E, Barranger JA. Cabrera-Salazar MA, et al. Curr Opin Mol Ther. 2002 Aug;4(4):349-58. Curr Opin Mol Ther. 2002. PMID: 12222873 Review.
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: cabrera salazar ma. PLoS One. 2011;6(6):e21758. doi: 10.1371/journal.pone.0021758. Epub 2011 Jun 29. PLoS One. 2011. PMID: 21738789 Free PMC article.