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AssigneeMount Sinai School of Medicine of the City University of New York
InventorsRobert J. Desnick, David F. Bishop, Yiannis A. Ioannou, Anne M. Wang
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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.
A method for producing human .alpha.-N-acetylgalactosaminidase, comprising:
A method for producing human .alpha.-N-acetylgalactosaminidase, comprising:
A recombinant vector comprising a nucleotide sequence encoding a cleavage site sandwiched between an .alpha.-N-acetylgalactosaminidase coding sequence and a protein A domain E coding sequence arranged in the same translational reading frame, controlled by a second nucleotide sequence that regulates the expression of a fusion protein in a mammalian host cell.
A recombinant vector pAGB-3, as deposited with the Agricultural Research Culture Collection having accession number B-18724.
A recombinant vector comprising a nucleotide sequence encoding a cleavage site sandwiched between an .alpha.-N-acetylgalactosaminidase coding sequence and a protein antigen coding sequence arranged in the same translational reading frame, controlled by a second nucleotide sequence that regulates the expression of a fusion protein in a mammalian host cell.
Dependencies are inferred from the claim text.