Preworkshop and Mini-Symposium for the Assessment of Transgenic Large Animals as Bioreactors to be held in Mountain Lake, Virginia, November 16-18, 1988

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The expression of mammalian proteins in conventional procaryotic and eukaryotic culture systems has many inadequacies. These shortcomings may preclude their successful utilization in bioreactors for the large-scale production of post-translational modified proteins. It is important to note that most human proteins possess a significant degree of post-translational modification. Even mammalian tissue culture systems which possess the necessary enzymatic machinery to make these modifications have limitations both with respect to inherent culture instability and expression levels. This is complicated by the high cost of culture media required for tissue culture. Preliminary research indicates that all of the above problems may be overcome by transgenic expression in large animals. An assessment of the current developments in basic transgenic biology is necessary such that its potential can be focused and advanced to become a part of the current gamut of emerging biotechnologies. This is a main goal of the workshop entitled, "Mt. Lake Va. Preworkshop and Minisymposium on Large Transgenic Animals as Bioreactors." The large-scale production of proteins which have therapeutic value is now performed for only a very few proteins. These proteins are either very simple molecules such as insulin or are effective in small quantities for one-time catastrophic events. This is the case for tissue plasminogen activator (TPA) which is a protein used in the treatment of a heart attack which is in progress. TPA is an illustrative example as its cost and limited availability are so prohibitive as to confine its use to (almost exclusively) emergency room situations| Proteins which are only effective as chronic therapies (ergo, on a long term basis) are not as a rule available in sufficient quantity or in a biologically active form as produced by current bioreactor technology. The use of transgenic cows as bioreactors may provide the solution to these protein production impasses through its unsurpassed ability to make protein in milk. Conservative estimates have put a single, average (transgenic) cow's annual production of any given important therapeutic protein in excess of tens of millions of dollars. This agricultural- biotechnological marriage would necessarily breathe new life into both the dairy and cattle industries as well as the people who now die as result of the unavailability of therapeutic proteins.