Deficiency of α-galactosidase A (α-GAL) causes Fabry disease (FD), an X-linked storage disease of the glycosphingolipid globtriaosylcerammide (Gb3) in lysosomes of various cells and elevated plasma globotriaosylsphingosine (Lyso-Gb3) toxic for podocytes and nociceptive neurons. Enzyme replacement therapy is used to treat the disease, but clinical efficacy is limited in many male FD patients due to development of neutralizing antibodies (Ab). Therapeutic use of modified lysosomal α-
Human Alpha Galactosidases Transiently Produced in
K. Kytidou, T. Beenakker, L. Westerhof, C. Hokke, G. Moolenaar, N. Goosen, M. Mirzaian, M. Ferraz, M. de Geus, W. Kallemeijn, H. Overkleeft, R. Boot, A. Schots, D. Bosch and J. Aerts
Front Plant Sci 2017;8:1026.