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. 2016 Apr 5;24(6):1019–1029. doi: 10.1038/mt.2016.53

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Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy

This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International License. The images or other third party material in this article are included in the article's Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/

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Effect of SRT on the extent of CD68 staining in the CNS of 4L;C* mice. CNS from 45-day-old untreated 4L/wild-type (4L/WT), vehicle-treated 4L;C* (4L;C*+ vehicle), and Genz-682452-treated 4L;C* (4L;C*+SRT) mice were subjected to immunohistochemical analysis to identify microglia (CD68 staining). Genz-682452 was dosed IP at 12.5 mg/kg/day from days 5 to 20 and at approximately 60 mg/kg/day in diet thereafter. Sections of spinal cord and brainstem are shown; brown coloration indicates positive staining for CD68 (a). Quantifications of percent area staining positively for CD68 in representative sections of the spinal cord, brainstem, and thalamus are shown for tissues taken at 21-, 35-, 45- and 65-days old (b). CNS, central nervous system; SRT, substrate reduction therapy.