Recombinant .alpha.-galactosidase a therapy for Fabry disease
AssigneeResearch Corporation Technologies, Inc.
InventorsDavid H. Calhoun, George Coppola
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Abstract
Fabry disease results from an X-linked deficiency in the enzyme .alpha.-galactosidase A. The present invention is directed to recombinant human .alpha.-galactosidase A and provides baculovirus expression vectors and recombinant virus that provide stable expression of extracellular and intracellular levels of this enzyme in an insect cell culture. The recombinant-derived enzyme can be used in enzyme replacement therapy to treat Fabry patients. Composition useful in therapeutic administration of .alpha.-galactosidase A are also provided.
Claims20
A replicable expression vector comprising a recombinant DNA or a cDNA encoding a precursor form of biologically active alpha-galactosidase A polypeptide which is operably linked to a baculovirus nucleic acid sequence.
A recombinant baculovirus comprising a nucleic acid encoding the precursor form of a biologically active .alpha.-galactosidase A.
A recombinant baculovirus comprising a nucleic acid encoding the mature form of a biologically active .alpha.-galactosidase A.
A recombinant baculovirus comprising a nucleic acid encoding biologically active .alpha.-galactosidase A having an amino acid sequence of ##STR4##
A recombinant baculovirus comprising a nucleic acid having a nucleotide sequence of ##STR5##
Dependencies are inferred from the claim text.