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

by Susanne Gustavsson, Elisabet Ohlin Sjöström, Agneta Tjernberg, Juliette Janson, Ulrica Westermark, Tommy Andersson, Åsa Makower, Erik Arnelöf, Gudrun Andersson, Jan Svartengren, Carina Ekholm, Stefan Svensson Gelius
Published 2019-12-01

Ancestral lysosomal enzymes with increased activity harbor therapeutic potential for treatment of Hunter syndrome

by Natalie M. Hendrikse, Anna Sandegren, Tommy Andersson, Jenny Blomqvist, Åsa Makower, Dominik Possner, Chao Su, Niklas Thalén, Agneta Tjernberg, Ulrica Westermark, Johan Rockberg, Stefan Svensson Gelius, Per-Olof Syrén, Erik Nordling
Published 2021-03-01