Impact of chemical modification of sulfamidase on distribution to brain interstitial fluid and to CSF after an intravenous administration in awake, freely-moving rats

by Juliette Janson, Gudrun Andersson, Lars Bergquist, Maria Eriksson, Joost H.A. Folgering
Published 2020-03-01

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