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Company Press Release Cell Genesys Scientists Demonstrate the Ability to Regulate EPO Gene Therapy FOSTER CITY, Calif., July 30 /PRNewswire/ -- Cell Genesys, Inc. today announced that company scientists have reported successful preclinical studies demonstrating the regulation of gene therapy. The ability to regulate the level of gene activity is an important feature for gene therapy applications that involve the delivery of a therapeutic protein such as erythropoietin (EPO), a protein deficient in patients with anemia. Following a single gene therapy treatment, the levels of EPO were increased or decreased by the systemic administration of tetracycline in studies in mice conducted over approximately 20 weeks. The genes for EPO and a tetracycline regulatory gene were injected directly into muscle utilizing two adeno-associated viral (AAV) gene delivery systems, or vectors. Cell Genesys scientists, led by Richard O. Snyder, Ph.D., reported this study in the August issue of the journal, Nature Biotechnology. ``Cell Genesys is the first to demonstrate that it is possible to regulate genes delivered by the AAV gene delivery system,'' said Mitchell H. Finer, Ph.D., vice president, research at Cell Genesys. ``This capability makes the AAV gene delivery system a potentially very attractive vehicle for the delivery of genes to treat disorders such as hemophilia, PARKINSON'S disease and anemia. Regulation of gene therapy could make this new modality for treating chronic diseases more practical by combining advantages of long-lasting treatments with ease of patient-specific therapy with oral drugs.'' EPO is a hormone which stimulates the production of red blood cells and is currently used in the treatment of anemia primarily resulting from kidney disease. A long lasting EPO gene therapy could potentially be administered to patients and regulated as needed. The ability to regulate an EPO gene therapy according to patient-specific requirements with the oral administration of tetracycline, further increases the attractiveness of a gene therapy treatment. In other studies, the company has demonstrated the effective delivery of a variety of genes utilizing AAV gene delivery vectors to the liver, the central nervous system and muscle tissue. Company scientists also have reported that following just a single injection of AAV gene therapy, production of EPO can be achieved in muscle for more than six months. Additionally, stable production of the enzyme responsible for the synthesis of L-dopa, a protein deficient in PARKINSON'S disease patients, can be achieved in the appropriate brain cells for at least one year. SOURCE: Cell Genesys, Inc. -- Judith Richards, London, Ontario, Canada [log in to unmask]