Metabolic rewiring and autophagy inhibition correct lysosomal storage disease in mucopolysaccharidosis IIIB

Metabolic rewiring and autophagy inhibition correct lysosomal storage disease in mucopolysaccharidosis IIIB

Summary: Mucopolysaccharidoses (MPSs) are lysosomal disorders with neurological involvement for which no cure exists. Here, we show that recombinant NK1 fragment of hepatocyte growth factor rescues substrate accumulation and lysosomal defects in MPS I, IIIA and IIIB patient fibroblasts. We investiga...

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Bibliographic Details
Main Authors: Melania Scarcella, Gianluca Scerra, Mariangela Ciampa, Marianna Caterino, Michele Costanzo, Laura Rinaldi, Antonio Feliciello, Serenella Anzilotti, Chiara Fiorentino, Maurizio Renna, Margherita Ruoppolo, Luigi Michele Pavone, Massimo D’Agostino, Valeria De Pasquale
Format: Article
Language:English
Published: Elsevier 2024-03-01
Series:iScience
Subjects:
Human metabolism
Cell biology
Online Access:http://www.sciencedirect.com/science/article/pii/S2589004224001809

Internet

http://www.sciencedirect.com/science/article/pii/S2589004224001809

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