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

Mucopolysaccharidosis type IIIA (MPS IIIA) is a lysosomal storage disorder (LSD) characterized by severe central nervous system (CNS) degeneration. The disease is caused by mutations in the SGSH gene coding for the lysosomal enzyme sulfamidase. Sulfamidase deficiency leads to accumulation of heparan...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6737345/
https://www.ncbi.nlm.nih.gov/pubmed/31528541
http://dx.doi.org/10.1016/j.ymgmr.2019.100510

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