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. 2023 Jan 11;30(6):487–502. doi: 10.1038/s41434-022-00381-y

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© The Author(s) 2023

Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.

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Fig. 6 — Data are mean ± SD, n = 4 animals per time point, analyzed 14 weeks after treatment at 7.5 months of age. n = 4 for untreated age-matched control group, n = 2 for WT mice. The relative reduction of Lyso-Gb3 is shown as % remaining storage. Unpaired t-test was used to compare means of all treatment groups. AAV8-FLT190 treated vs. untreated controls: plasma Gb3 (p = 0.0084); plasma Lyso-Gb3 (p < 0.0001); urine Gb3 (p = 0.02); kidney Gb3 (p = 0.045); kidney Lyso-Gb3 (p = 0.022); heart Gb3 (p < 0.0001); heart Lyso-Gb3 (p ≤ 0.0001); liver Gb3 (p = 0.0075). AAV8 = adeno-associated virus serotype 8; lyso-Gb3 = globotriaosylsphingosine; Gb3 = globotriaosylceramide; SD = standard deviation; vg = vector genomes; WT = wild type.