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

1994-06-17

1996-12-03

Wax, Robert A.

Chemistry: molecular biology and microbiology

Micro-organism, tissue cell culture or enzyme using process...

Recombinant dna technique included in method of making a...

435208, 4353201, 935 47, C12P 2106, C12N 1562, C12N 940

Patent

active

055807577

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.

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