Abstract: The invention provides transgenic nonhuman mammals producing phosphorylated lysosomal proteins in their milk, and methods of generating the same. Phosphorylation occurs at the 6' position of a mannose side chain residue. Also provided are methods of purifying lysosomal proteins from milk, and incorporating the proteins into pharmaceutical compositions for use in enzyme replacement therapy.
Type:
Grant
Filed:
July 29, 1996
Date of Patent:
September 12, 2000
Assignees:
Pharming B.V., The Universiteit Leiden, Academic Hospital, Eramus Universiteit
Inventors:
Arnold J. J. Reuser, Ans T. Van der Ploeg, Frank R. Pieper, Martin Ph. Verbeet