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

Autor: Lund TC; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota, Minneapolis, Minnesota, USA., Braunlin E; Department of Pediatrics, Division of Pediatric Cardiology, University of Minnesota, Minneapolis, Minnesota, USA., Polgreen LE; The Lundquist Institute at Harbor-UCLA Medical Center, Torrance, California, USA., Gupta AO; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota, Minneapolis, Minnesota, USA., Orchard PJ; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota, Minneapolis, Minnesota, USA., Eisengart JB; Department of Pediatrics, Division of Clinical Behavioral Neuroscience, University of Minnesota, Minneapolis, Minnesota, USA.
Jazyk: angličtina
Zdroj: Annals of neurology [Ann Neurol] 2023 Dec; Vol. 94 (6), pp. 1182-1186. Date of Electronic Publication: 2023 Sep 23.
DOI: 10.1002/ana.26786
Abstrakt: 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 naïve (n = 19) versus receiving standard non-BBB penetrating enzyme replacement therapy (ERT, n = 12). In the ERT versus treatment naïve 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;94:1182-1186.
(© 2023 The Authors. Annals of Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.)
Databáze: MEDLINE
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