ENZYME REPLACEMENT THERAPY FOR TREATING MPS VII RELATED BONE LESIONS USING A CHEMICALLY MODIFIED ENZYME

摘要

The invention relates to a method of treating mucopolysaccharidoses using enzyme replacement therapy with chemically modified lysosomal enzymes. More specifically the method relates to administering chemically modified lysosomal enzymes intraperitoneal injection. In addition, the invention relates to treating type VII mucopolysaccharidoses or mucopolysaccharidoses type VII related bone lesions with a chemical modified β-glucuronidase, wherein the modified β-glucuronidase may be administered 5 weeks after birth, and or may be administered intraperitoneally.

主权项

1. A method of administering enzyme replacement therapy to a subject with an lysosomal enzyme deficiency, the method comprising:
a) modifying a lysosomal enzyme determined to be deficient in the subject with sodium meta-periodate treatment followed by treatment with sodium borohydride, wherein clearance from blood is prolonged, and enzyme activity is retained; and b) administering an effective amount of the modified lysosomal enzyme by intraperitoneal injection.