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Gene therapy for lysosomal storage disorders.
Ioannou YA, Enriquez A, Benjamin C. Ioannou YA, et al. Expert Opin Biol Ther. 2003 Aug;3(5):789-801. doi: 10.1517/14712598.3.5.789. Expert Opin Biol Ther. 2003. PMID: 12880379 Review.
An "exacerbate-reverse" strategy in yeast identifies histone deacetylase inhibition as a correction for cholesterol and sphingolipid transport defects in human Niemann-Pick type C disease.
Munkacsi AB, Chen FW, Brinkman MA, Higaki K, Gutiérrez GD, Chaudhari J, Layer JV, Tong A, Bard M, Boone C, Ioannou YA, Sturley SL. Munkacsi AB, et al. Among authors: ioannou ya. J Biol Chem. 2011 Jul 8;286(27):23842-51. doi: 10.1074/jbc.M111.227645. Epub 2011 Apr 13. J Biol Chem. 2011. PMID: 21489983 Free PMC article.
149 results