Pentosan Polysulfate Treatment of Mucopolysaccharidosis Type IIIA Mice

戊聚糖多硫酸盐治疗粘多糖贮积症 IIIA 型小鼠

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作者:Ningning Guo, Victor DeAngelis, Changzhi Zhu, Edward H Schuchman, Calogera M Simonaro

Results

As seen in other MPS animal models, subQ PPS treatment reduced plasma cytokine levels and macrophage infiltration in systemic tissues. ICV administration did not elicit these systemic effects. SubQ PPS administration also significantly impacted brain neuropathology, inflammation, and behavior. The effect of early subQ treatment was more significant than dose. Surprisingly, ICV PPS treatment had intermediate effects on most of these brain markers, perhaps due to the limited dose and/or duration of treatment. Consistent with these neuropathological findings, we also observed significant improvements in the hyperactivity/anxiety and learning behaviors of the MPS IIIA mice treated with early subQ PPS.

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