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FOR IMMEDIATE RELEASE |
CONTACT: Michelle Reardon |
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Monday, March 27, 2000 |
302/577-8701 |
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Boston, MA - Molecular biologist Dr. Eric B. Kmiec, widely regarded worldwide for his work in the field of DNA repair and responsible for developing a technique for repairing disease-causing genetic mutations, presented some of his recent findings on functional genomics at the world's premiere biotechnology symposium today. Kmiec presented information about the his latest research to identify genes that have no known genetic components, but contain genetic information that provide a clue to the make-up, and thus cure of viruses. His studies have shown that these gene components can be used as a test system to screen for new therapeutics. This new scientific development can be applied to all genetic applications including crop research, human disease and clinical drug development. Kmiec, also Director of the Laboratory of Genetic Medicine and Genomics at the University of Delaware, recently moved his lab to the University of Delaware (UD) from Thomas Jefferson University this past fall. The gene therapy lab is an affiliation of the Delaware Biotechnology Institute at UD -- a partnership among government, industry and academia designed to position Delaware as a center of excellence in biotechnology and life sciences. Author of the landmark "350 patent," covering a specially engineered molecule that fixes "snips" (single nucleotide polymorphisms) in DNA, Kmiec has focused his research on sickle-cell anemia, Huntington's Disease and food-crop advances. Kmiec credits his then-three-year-old son, Tyler, now 8, with inspiring him to develop a molecule that acts like nature's seamstress, by stitching mutated genetic sequences back together in their proper order. As Kmiec tells it, Tyler was sitting on his father's lap as they rode a tractor-mower. After hearing his father's detailed description of genetic defects, Kmiec says, his son advised him to "just put things together." The result was a chemically grafted, or "chimeric" molecule composed of DNA and small amounts of RNA, which seeks out mismatched strands of DNA. When a mutated gene is identified, the chimeric molecule then triggers the cell's natural repair system to replace the chemical bases in question. Researchers worldwide are conducting promising investigations of such diseases as muscular dystrophy using Kmiec's technique. Kmiec and his wife, Jennifer, cofounded Kimeragen, the Newtown, PA company now gearing up for a human clinical trial of Kmiec's gene-repair technique. In the August 31, 1999 Proceedings of the National Academy of Sciences, Kimeragen researchers reported that they used Kmiec's technique on rats to correct the mutation responsible for Crigler-Najjar syndrome, which causes the toxic bile component, bilirubin, to accumulate in the body. From Human Disease to Crop Research "Sickle-cell anemia is a terrible disease," Kmiec says of the painful group of hereditary red blood disorders caused by abnormal, sickle-shaped blood cells." Unfortunately, patients are faced with few alternative treatment options. University of Delaware senior scientist Allyson Cole-Strauss developed the first-of-its-kind bench top method for studying gene repair in cells. This technology set the stage for Kmiec's research. She is now leading the sickle-cell anemia investigation at Delaware. Building on previous cellular studies, the team is working to examine the system's effectiveness in mice, in collaboration with doctors from the A.I. duPont Hospital for Children. More nutritious, disease-resistant food crops could someday result from Kmiec's research. By shooting chimeric molecules into plant genes, it may be possible to alter the genetic information that makes plants vulnerable to herbicides, rot and other maladies. The technique should be more precise than traditional plant-breeding techniques, Kmiec says, and less controversial than "transgenic" strategies, which involve introducing foreign DNA into plant genes. Kmiec says he remains "cautiously optimistic" about his gene-repair technique, though he emphasizes the need for additional studies of fundamental questions. "Researchers in this field are doing excellent, promising work," he says. "Today the real danger is in a public negative reaction that could halt the process of scientific discovery in the field of genetic medicine. This should not be allowed to happen." More than 7,000 company executives, scientists, investment experts and government officials from over 40 nations are attending BIO 2000, the world's premiere biotechnology conference, from March 27 through March 30 in Boston, Massachusetts. More than 600 speakers are discussing the latest developments in biotechnology science, business and public policy. |
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