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Last Updated: March 27, 2026

Claims for Patent: 5,382,524


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Summary for Patent: 5,382,524
Title:Cloning and expression of biologically active α-n-acetylgalactosaminidase
Abstract: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.
Inventor(s):Robert J. Desnick, David F. Bishop, Yiannis A. Ioannou, Anne M. Wang
Assignee: Icahn School of Medicine at Mount Sinai
Application Number:US07/602,608

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