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

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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Autori principali: Pascale Tuyaa-Boustugue, Ingrid Jantzen, Haoyue Zhang, Sarah P. Young, Pierre Broqua, Mireille Tallandier, Eugeni Entchev
Natura: Articolo
Lingua:English
Pubblicazione: Elsevier 2023-12-01
Serie:Molecular Genetics and Metabolism Reports
Soggetti:
Mucopolysaccharidosis type VI
Mucopolysaccharidosis type I
Lysosome
Odiparcil
MPS VI therapy
MPS I therapy
Accesso online:http://www.sciencedirect.com/science/article/pii/S2214426923000575

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http://www.sciencedirect.com/science/article/pii/S2214426923000575

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