Hurler Syndrome Glycosaminoglycans Decrease in Cerebrospinal Fluid without Brain-Targeted Therapy

Troy C. Lund

doi:10.1002/ana.26786

2023-09-23

DOI：10.1002/ana.26786

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摘要

En 中文

Novel therapies for Hurler syndrome aim to cross the blood-brain barrier (BBB) to target neurodegeneration by degrading glycosaminoglycans (GAG). BBB penetration has been assumed with decreased cerebrospinal fluid (CSF) GAG, yet little is known about CSF GAG without brain-targeting therapies. We compared pre-transplant CSF GAG in patients who were treatment naive (n = 19) versus receiving standard non-BBB penetrating enzyme replacement therapy (ERT, n = 12). In the ERT versus treatment naive groups, CSF GAG was significantly lower across all content assayed, raising questions about using CSF GAG decrements to show BBB penetration. Future studies should compare GAG reduction in standard versus novel therapies. ANN NEUROL 2023

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DISEASE

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期刊

Annals of Neurology

IF:

7.7

论文数:

8.5K

被引数:

3.6W

机构

University of Minnesota Twin Cities

学者数:

3.7W

论文数: 3.1W

被引数: 58

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