Methods and compositions for treatment of forbes-cori disease
AssigneeValerion Therapeutics, LLC
InventorDustin D. Armstrong
Life of this patent
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Abstract
In certain embodiments, the present disclosure provides compositions and methods for treating Forbes-Cori Disease.
Claims33
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A method of treating Forbes-Cori disease in a subject in need thereof, comprising contacting the cell with a chimeric polypeptide comprising:
- (i) a mature acid alpha-glucosidase (GAA) polypeptide and (ii) an internalizing moiety that promotes delivery into cells;
- wherein the chimeric polypeptide has acid alpha-glucosidase activity, and wherein the chimeric polypeptide does not comprise a GAA precursor polypeptide of approximately 110 kilodaltons.
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A method of decreasing glycogen accumulation in cytoplasm of cells of a Forbes-Cori patient, comprising contacting muscle cells with a chimeric polypeptide, which chimeric polypeptide comprises (i) a mature acid alpha-glucosidase (GAA) polypeptide and (ii) an internalizing moiety that promotes transport into cytoplasm of cells;
- wherein the chimeric polypeptide has acid alpha-glucosidase activity, and wherein the chimeric polypeptide does not comprise a GAA precursor polypeptide of approximately 110 kilodaltons.
A method of increasing GAA activity in the cytoplasm of a cell, comprising delivering a chimeric polypeptide, wherein said chimeric polypeptide comprises:
- (i) a mature acid alpha-glucosidase (GAA) polypeptide and (ii) an internalizing moiety that promotes transport into cytoplasm of cells;
- wherein the chimeric polypeptide has acid alpha-glucosidase activity, and wherein the chimeric polypeptide does not comprise a GAA precursor polypeptide of approximately 110 kilodaltons.
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Dependencies are inferred from the claim text.