Fig. 5: MG01 prevents GAG accumulation in the CNS of MPS I and MPS VII mouse models at 1-, 5- or 8-months post-treatment.

A Schematic depicting experimental design of the initial experiment in MPS I mice with Line 6-derived MG01. B Accumulation of GAGs in the brain of adult-treated MPS I cohorts at 4-months post-treatment with Line 6-derived MG01. C Schematic depicting the experimental design of adult (MPS I: P60; MPS VII: P90) and juvenile (P21) MG01 transplantation in MPS I and MPS VII mouse models. D Accumulation of GAGs at 1-, 5- and 8-months post-treatment in the brain, spinal cord (SpC), or CSF of the juvenile-treated MPS I cohorts. MG01 was administered at a low (0.4 × 10⁶) or high dose (1.4 × 10⁶). E Correlation between CSF and brain GAG levels at 5 months post-treatment in the juvenile MPS I cohort. F Accumulation of GAGs at 1- and 5-months post-treatment in the brain, spinal cord, or CSF of the adult-treated MPS I cohorts. G Accumulation of GAGs at 1-month post-treatment in the brain and spinal cord of the juvenile-treated MPS VII cohorts. H Accumulation of GAGs at 1- and 5-months post-treatment in the brain and spinal cord of the adult-treated MPS VII cohorts. GAG levels were measured as μg/ml and are depicted as percentage of GAGs in vehicle controls of MPS I or MPS VII, respectively. Significance was tested with a one-way ANOVA using Dunnet’s multiple comparison test. Graphs denote mean +/− SEM. Data points in B, D, and H represent individual animals. (n.s.: not significant, *p ≤ 0.05, **p ≤ 0.01, ***p ≤ 0.001, ****p ≤ 0.0001). Panels A, and C were in part created with BioRender.com released under a Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International license.