Chemically modified cystathionine beta-synthase enzyme for treatment of homocystinuria
AssigneeThe Regents of the University of Colorado, a body corporate
InventorsJan P Kraus, Tomas Majtan, Erez Bublil
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Abstract
The invention provides reagents and methods for enzyme replacement therapy using chemically modified species of human cystathionine β-synthase (CBS) to treat homocystinuria and other related diseases and disorders.
Claims17
A method for treating or ameliorating a disease, disorder, or condition associated with elevated homocysteine, the method comprising administering a composition comprising an isolated carboxyl-terminal truncated cystathionine-β-synthase (CBS) polypeptide, said truncated CBS polypeptide comprising a carboxyl-terminal truncation spanning any of amino acid residues 383-551 of SEQ ID NO:
- 2, wherein said truncated CBS polypeptide is a chemically cleaved, enzymatically cleaved or genetically engineered truncate thereof, and wherein said truncated CBS polypeptide is covalently linked to one or a plurality of polyethylene glycol molecules.
A method for preventing a disease, disorder, or condition associated with elevated homocysteine, the method comprising administering a composition comprising an isolated carboxyl-terminal truncated cystathionine-β-synthase (CBS) polypeptide, said truncated CBS polypeptide comprising a carboxyl-terminal truncation spanning any of amino acid residues 383-551 of SEQ ID NO:
- 2, wherein said truncated CBS polypeptide is a chemically cleaved, enzymatically cleaved or genetically engineered truncate thereof, and wherein said truncated CBS polypeptide is covalently linked to one or a plurality of polyethylene glycol molecules.
A composition comprising an isolated carboxyl-terminal truncated cystathionine-β-synthase (CBS) polypeptide, said truncated CBS polypeptide comprising a carboxyl-terminal truncation spanning any of amino acid residues 383-551 of SEQ ID NO:
- 2, wherein said truncated CBS polypeptide is a chemically cleaved, enzymatically cleaved or genetically engineered truncate thereof, that is covalently linked to one or a plurality of polyethylene glycol molecules.
Dependencies are inferred from the claim text.