Ontology highlight
ABSTRACT:
SUBMITTER: Tian W
PROVIDER: S-EPMC6491494 | biostudies-literature | 2019 Apr
REPOSITORIES: biostudies-literature
Tian Weihua W Ye Zilu Z Wang Shengjun S Schulz Morten Alder MA Van Coillie Julie J Sun Lingbo L Chen Yen-Hsi YH Narimatsu Yoshiki Y Hansen Lars L Kristensen Claus C Mandel Ulla U Bennett Eric Paul EP Jabbarzadeh-Tabrizi Siamak S Schiffmann Raphael R Shen Jin-Song JS Vakhrushev Sergey Y SY Clausen Henrik H Yang Zhang Z
Nature communications 20190430 1
Lysosomal replacement enzymes are essential therapeutic options for rare congenital lysosomal enzyme deficiencies, but enzymes in clinical use are only partially effective due to short circulatory half-life and inefficient biodistribution. Replacement enzymes are primarily taken up by cell surface glycan receptors, and glycan structures influence uptake, biodistribution, and circulation time. It has not been possible to design and systematically study effects of different glycan features. Here w ...[more]