Transgenic Cloned Goats and the Production of Recombinant Therapeutic Proteins

The clinical use of recombinant therapeutic proteins has increased significantly during the last few decades. However, therapeutic indications often require large amounts of highly purified product, especially for therapies that call for chronic dosing regimes. The development of very efficient expression systems is essential to the full exploitation of the recombinant technology, so that life-saving medicines will become more readily available. Transgenic production technology is a highly scalable, capital-sparing approach to the manufacturing of complex recombinant proteins. ATryn®, recombinant antithrombin purified from the milk of transgenic goats, the first transgenically-derived therapeutic protein to gain regulatory approval, is currently approved in the United States and the EU for the treatment of Hereditary Antithrombin Deficiency. In addition, production of additional recombinant proteins in the milk of transgenic goats and rabbits is currently being tested for the production of a number of therapeutic antibodies as well as an alternative to plasma fractionation for the manufacture of human plasma proteins. The following review examines the potential of somatic cell nuclear transfer to generate transgenic goats used in the production of human recombinant therapeutics.