Intravenous delivery of a chemically modified sulfamidase efficiently reduces heparan sulfate storage and brain pathology in mucopolysaccharidosis IIIA mice-Reference-Cited by-同舟云学术

Intravenous delivery of a chemically modified sulfamidase efficiently reduces heparan sulfate storage and brain pathology in mucopolysaccharidosis IIIA mice

Published:2019-12 Issue: Volume:21 Page:100510
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Gustavsson Susanne,Ohlin Sjöström Elisabet,Tjernberg Agneta,Janson Juliette,Westermark Ulrica,Andersson Tommy,Makower Åsa,Arnelöf Erik,Andersson Gudrun,Svartengren Jan,Ekholm Carina,Svensson Gelius Stefan

Funder

Swedish Orphan Biovitrum AB

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference46 articles.

1. Mucopolysaccharide diseases: a complex interplay between neuroinflammation, microglial activation and adaptive immunity;Archer;J. Inherit. Metab. Dis.,2014

2. Early neurodegeneration progresses independently of microglial activation by heparan sulfate in the brain of mucopolysaccharidosis IIIB mice;Ausseil;PLoS One,2008

3. Axonal dystrophy in the brain of mice with Sanfilippo syndrome;Beard;Exp. Neurol.,2017

4. A novel missense mutation in lysosomal sulfamidase is the basis of MPS III A in a spontaneous mouse mutant;Bhattacharyya;Glycobiology,2001

5. A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome);Bhaumik;Glycobiology,1999

Cited by 17 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic Mucopolysaccharidoses;Molecular Genetics and Metabolism;2024-08

2. Engineered arylsulfatase A with increased activity, stability and brain delivery for therapy of metachromatic leukodystrophy;Molecular Therapy;2023-10

3. Neurocognitive testing in a murine model of mucopolysaccharidosis type IIIA;Molecular Genetics and Metabolism Reports;2023-09

4. Advances in therapies for neurological lysosomal storage disorders;Journal of Inherited Metabolic Disease;2023-05-02

5. Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier;Molecular Therapy;2023-03