Reduction of lysosome abundance and GAG accumulation after odiparcil treatment in MPS I and MPS VI models

Deficiencies of lysosomal enzymes responsible for the degradation of glycosaminoglycans (GAG) cause pathologies commonly known as the mucopolysaccharidoses (MPS). Each type of MPS is caused by a deficiency in a specific GAG-degrading enzyme and is characterized by an accumulation of disease-specific...

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Main Authors: Pascale Tuyaa-Boustugue, Ingrid Jantzen, Haoyue Zhang, Sarah P. Young, Pierre Broqua, Mireille Tallandier, Eugeni Entchev
格式: Article
語言:English
出版: Elsevier 2023-12-01
叢編:Molecular Genetics and Metabolism Reports
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在線閱讀:http://www.sciencedirect.com/science/article/pii/S2214426923000575