Cloning and expression of biologically active .alpha.-galactosid

Details Cloning and expression of biologically active .alpha.-galactosid Cloning and expression of biologically active .alpha.-galactosid

1992-11-30

1995-03-28

Wax, Robert A.

Chemistry: molecular biology and microbiology

Enzyme , proenzyme; compositions thereof; process for...

Hydrolase

435193, 935 14, 935 47, 536 232, 536 234, C12N 940, C12N 910, C12N 1500

Patent

active

054016509

ABSTRACT:
The present invention involves the production of large quantities of human .alpha.-Gal A by cloning and expressing the .alpha.-Gal A coding sequence in eukaryotic host cell expression systems. The eukaryotic expression systems, and in particular the mammalian host cell expression system described herein provide for the appropriate cotranslational and posttranslational modifications required for proper processing, e.g., glycosylation, phosphorylation, etc. and sorting of the expression product so that an active enzyme is produced. In addition, the expression of fusion proteins which simplify purification is described.
Using the methods described herein, the recombinant .alpha.-Gal A is secreted by the engineered host cells so that it is recovered from the culture medium in good yield. The .alpha.-Gal A produced in accordance with the invention may be used, but is not limited to, in the treatment in Fabry Disease; for the hydrolysis of .alpha.-galactosyl residues in glycoconjugates; and/or for the conversion of the blood group B antigen on erythrocytes to the blood group O antigen.

REFERENCES:
patent: 3966555 (1976-06-01), Arnaud et al.
patent: 3972777 (1976-08-01), Yamada et al.
patent: 4450238 (1984-05-01), Vitobelko et al.
Bishop et al., 1985, Am. J. Hum Genetics 37 (4 Suppl) A 144.
Calhoun et al., 1985, Proc. Natl. Acad. Sci. USA 82: 7364-73698.
Bishop et al., 1986, Proc. Natl. Acad. Sci. USA 83: 4859-4863.
Hantzopoulos & Calhoun, 1987, Gene (Amst.) 57: 159-169.
Quinn et al., 1987, Gene (Amst.) 58: 177-188.
Tsuji et al., 1987, Eur. J. Biochem. 165(2): 275-280.
Bishop et al., 1988, Proc. Natl. Acad. Sci. USA 85: 3903-3907.
Bishop et al., 1988, in Lipid Storage Disorders, Eds. Salvayre et al., Plm Publishing Corp., pp. 809-822.
Kornreich et al., 1989, Nuc. Acids Res. 17(8): 3301-3302.
Ioannou et al., Jun. 1990, in Inborn Errors of Metabolism, Vth International Congress, Abstract OC4.3.
Coppola et al., 1989, Biotechnology and Human Genetic Predisposition to Disease, J. Cell Biochem. Suppl., Abstract K306.
Brady et al., 1973, N. Engl. J. Med. 289(1): 9-14.
Desnick, et al. 1979, Proc. Natl. Acad. Sci. USA 76: 5326-5330.
Desnick et al., 1980, Birth Defects: Original Article Series, XVI(1): 393-413.
Bishop et al., 1981, in Lysosomes and Lysosmal Storage Diseases, Eds. Callahan et al., Raven Press, pp. 381-394.
Hasholt & Sorenson, 1986, Human Genet. 72: 72-76.
Lemansky et al., 1987, J. Biol. Chem. 262(5): 2062-2065.
Desnick et al., 1987, Enzyme 38: 54-64.
Bernstein, et al., 1989, J. Clin. Invest. 83: 1390-1399.
Kornreich, et al. 1990, J. Biol. Chem. 265(16): 9319-9326.
Bishop et al., 1980, Birth Defects: Original Article Series, XVI(1): 17-32.
Bishop & Desnick, 1981, J. Biol. Chem. 256(3): 1307-1316.
Dean, K. J. et al., 1977, Biochem. Biophys. Res. Commun. 77(4): 1411-1417.
Schram, A. W. et al., 1977, Biochimica et Biophysica Acta 482: 138-144.
Sweeley, C. C. et al., 1983, Archives of Biochemistry and Biophysics 223(1): 158-65.
Tsuji, S. et al., 1989, Biochem. Biophys. Res. Commun. 163(3): 1498-1504.
Yamauchi, T. et al., 1990, Biochem. Biophys. Res. Commun. 170(1): 231-237.
Wang, A. M. et al., 1990, J. Biol. Chem. 265(35): 21859-21866.
Wang, A. M. et al., 1988, Am. J. Hum. Genet. 43(3 Suppl.): A99.
Schindler, D. et al., Jun. 29, 1989, New Engl. J. Med. 320(26): 1735-1740.
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.
Wang, A. M. et al., 1989, Am. J. Hum. Genet. 45(4 Suppl.): A228.
Wang, A. M. et al., 1990, J. Clin. Invest. 86: 1752-1756.
Desnick, R. J. and Wang, A. M., 1990, J. Inher. Metab. Dis. 13: 549-559.
Beutler, E. and Kuhl, W., 1972, J. Biol. Chem. 247(22): 7195-7200.
Callahan, J. W. et al., 1973, Biochemical Medicine 7: 424-431.
Kusiak, J. W. et al., 1978, J. Biol. Chem. 253(1): 184-190.
Dean, K. J. and Sweeley, C. C., 1979, J. Biol. Chem. 254(20): 10001-10005.
Bishop, D. F. et al., 1980, in Birth Defects: Original Article Series, XVI(1): 17-32 Desnick, R. J. et al., Editors Alan R. Liss, Inc., New York.
Weinstein et al., 1987, J. Biol. Chem. 262: 17735.

Affiliated with

Also associated with

Rating

Cloning and expression of biologically active .alpha.-galactosid does not yet have a rating. At this time, there are no reviews or comments for this patent.

If you have personal experience with Cloning and expression of biologically active .alpha.-galactosid, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Cloning and expression of biologically active .alpha.-galactosid will most certainly appreciate the feedback.

Rate now

Comments { 0 }

Profile ID: LFUS-PAI-O-2249927