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US 12,565,643 B2Granted
Alpha-galactosidase protein for enzyme replacement therapy (ERT) and methods of use
AssigneeThe Medical College of Wisconsin, Inc.
InventorsJeffrey A. Medin, Simone Scalia
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Abstract
The present disclosure provides less immunogenic mutant α-galactosidase A protein (α-gal), methods of making and methods of use. The less immunogenic mutant α-gal of the present invention provides a reduced immune response when administered to a subject.
Claims5
A virus comprising an expression construct comprising a nucleic acid sequence encoding a mutant α-galactosidase A protein (α-gal), wherein the mutant α-gal comprises a S62N amino acid change, wherein the amino acid change is relative to SEQ ID NO:
- 1, wherein the mutant a-gal has at least 98% identity to SEQ ID NO:
- 1, and wherein the virus is a retrovirus, adenovirus, or adeno-associated virus.
Dependencies are inferred from the claim text.