Cloning and expression of biologically active alpha -n-acetylgalactosaminidase

摘要

The present invention involves the production of human alpha -GalNAc by cloning and expressing the alpha -GalNAc coding sequence in eukaryotic host cell expressions systems. The eukaryotic expression systems, and in particular the mammalian host cell expression systems described herein provide for the appropriate co-translational and post-translation modifications required or proper processing, e.g., glycosylation, phosphorylation, etc. and sorting of the expression product so that an active enzyme is produced. The alpha -GalNAc produced in accordance with the invention may be used in the treatment of Schindler disease or for the hydrolysis of alpha -N-acetylgalactosaminyl moieties in various glycoconjugates.