Recombinant alpha-galactosidase a therapy for Fabry disease

摘要

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.