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US 2007/0011754 A1Published application
Lysosomal proteins produced in the milk of transgenic animals
OwnerArnold Reuser, Ans T. Ploeg +2inventors
InventorsArnold Reuser, Ans T. Ploeg, Frank Pieper, Martin Verbeet
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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.
Claims20
A transgenic nonhuman mammal having a transgene comprising:
- a mammary-gland specific promoter;
- a mammary-gland specific enhancer;
- a secretory DNA segment encoding a signal peptide functional in mammary secretory cells of the transgenic nonhuman mammal;
- and
- a recombinant DNA segment encoding a lysosomal protein operably linked to the secretory DNA segment to form a secretory-recombinant DNA segment, the secretory-recombinant DNA segment being operably linked to the promoter and to the enhancer;
- wherein the transgene, in an adult form of the nonhuman mammal or a female descendent of the nonhuman mammal, is capable of expressing the secretory-recombinant DNA segment in the mammary secretory cells to produce a form of the lysosomal protein that is process and secreted by the mammary secretory cells into milk as a mannose 6-phosphate containing lysosomal protein.
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Dependencies are inferred from the claim text.