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The Whey Acidic Protein (WAP) Promoter and Its Use to Express Therapeutic Proteins in the Milk of Transgenic Mammals

Posted Jun 15 2010 5:00pm

Description of Invention:
Transgenic animals can be engineered to express complex human proteins at high concentrations in milk. Protein replacement therapy is often the only treatment available for congenital diseases such as hemophilia or lysosomal storage disease, and the cost of treatment can be high with the therapeutic protein market estimated to reach more than $50 billion by 2010.

U.S. Patent No. 6,727,405 provides for a non-human mammal such as mouse, sheep, pig, goat and cow whose genome contains a DNA sequence comprising a milk serum protein (whey acidic protein) promoter linked to a heterologous gene sequence and secretory peptide, as well as methods for producing a secreted protein into the transgenic animal’s milk and claims directed to the DNA construct. The invention permits the production of any desired protein in an easily maintained, stable, mammalian bioreactor, which is capable not only of producing the desired protein in milk, but can also pass the ability to do so to its female offspring. Although other methods of obtaining recombinant protein products are available, these require inefficient, expensive purification of the protein from the blood or from cell culture media and there remains a need for an efficient and cost effective method for producing therapeutic proteins.

This WAP promoter platform technology provides a viable alternative to other milk protein promoters.

Lothar G Hennighausen (NIDDK)
Heinrich J Westphal (NICHD)

Patent Status:
HHS, Reference No. E-411-1987/0
US, , Patent No. 6,727,405, Issued 27 Apr 2004

Licensing Status:
Available for licensing.

Gene Based Therapies
Gene Based Therapies - Other
Animal Model

For Additional Information Please Contact:
Suryanarayana Vepa Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-435-5020
Fax: 301-402-0220

Ref No: 1026

Updated: 06/2010

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