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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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Detalles Bibliográficos
Autores principales: Tuyaa-Boustugue, Pascale, Jantzen, Ingrid, Zhang, Haoyue, Young, Sarah P., Broqua, Pierre, Tallandier, Mireille, Entchev, Eugeni
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10694777/
http://dx.doi.org/10.1016/j.ymgmr.2023.101011