Chemistry: molecular biology and microbiology – Enzyme – proenzyme; compositions thereof; process for... – Hydrolase
Patent
1990-10-24
1995-01-17
Wax, Robert A.
Chemistry: molecular biology and microbiology
Enzyme , proenzyme; compositions thereof; process for...
Hydrolase
435208, 4351721, 4352523, C12N 1500, C12N 940, C12N 924
Patent
active
053825241
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.
REFERENCES:
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Desnick, R. J. et al., 1989, in The Metabolic Basis of Inherited Disease, Scriver, C. R., et al., eds. Ch. 70 entitled "Fabry Disease: .alpha.-Galactosidase Deficiency; Schindler Disease: .alpha.-N-Acetylgalactosaminidase Deficiency", pp. 1751-1796, McGraw Hill, New York.
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Bishop David F.
Desnick Robert J.
Ioannou Yiannis A.
Wang Anne M.
Hendricks Keith D.
The Mount Sinai School of Medicine of the City University of New
Wax Robert A.
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