Crossing the gates of Babylon: Brain-penetrating enzyme replacement for lysosomal disorders
Author:
Funder
CNPq
FAPERGS
Publisher
Elsevier BV
Subject
Genetics,Molecular Biology,Molecular Medicine
Reference5 articles.
1. Mucopolysaccharidoses and the blood-brain barrier;Sahin;Fluids Barriers CNS,2022
2. Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study;Muenzer;Mol. Genet. Metab.,2022
3. Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study;Chen;Mol. Genet. Metab.,2020
4. Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice;Kida;Mol Ther Methods Clin Dev,2023
5. Evaluation of cerebrospinal fluid heparan sulfate as a biomarker of neuropathology in a murine model of mucopolysaccharidosis type II using high-sensitivity LC/MS/MS;Tanaka;Mol. Genet. Metab.,2018
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