In vivo brain delivery of BBB-enabled iduronate 2-sulfatase in rats

In vivo brain delivery of BBB-enabled iduronate 2-sulfatase in rats

Abstract Background Iduronate-2-sulfatase (IDS) deficiency (MPS II; Hunter syndrome) is a disorder that exhibits peripheral and CNS pathology. The blood brain barrier (BBB) prevents systemic enzyme replacement therapy (ERT) from alleviating CNS pathology. We aimed to enable brain delivery of systemi...

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Main Authors: Will J. Costain, Arsalan S. Haqqani, Greg Hussack, Henk van Faassen, Etienne Lessard, Binbing Ling, Eric Brunette, Dao Ly, Hung Fang, Jennyfer Bultinck, Steven Geysens, Gwenda Pynaert, Kathleen Piens, Stefan Ryckaert, Franck Fudalej, Wouter Vervecken, Danica Stanimirovic
Format: Article
Language:English
Published: BMC 2025-01-01
Series:Fluids and Barriers of the CNS
Subjects:
Blood brain barrier
MPS II
Iduronate-2-sulfatase
Enzyme replacement therapy
CSF
Brain
Online Access:https://doi.org/10.1186/s12987-024-00617-6
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