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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Foilsithe in:Mol Genet Metab Rep
Main Authors: 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
Formáid: Artigo
Teanga:Inglês
Foilsithe: Elsevier 2019
Ábhair:
Research Paper
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC6737345/
https://ncbi.nlm.nih.gov/pubmed/31528541
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2019.100510
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