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. 2023 Apr 12;14:2057. doi: 10.1038/s41467-023-37632-4

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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 — a PK study in Gba+/+ mice to assess systemic exposure of mGCase-mBS in plasma and brain. n = 3 mice/group. b Multi-dose PD study in 4 L/PS-NA mice to compare equimolar doses of mGCase vs. mGCase-mBS. GlcSph levels were measured in cortex, midbrain and liver. n = 6 mice/group. Data are represented as group mean +/− SEM. Data was analysed by one-way ANOVA (Dunnett’s multiple comparisons test) comparing each treatment group to 4 L/PS-NA, vehicle. n.s. p > 0.05; ****p < 0.0001. Source data are provided as a Source Data file. Schematics created with BioRender.com.