Apr. 2, 2002 MINNEAPOLIS / ST. PAUL (April 1, 2002)-- For the first time, a special segment of DNA called a transposon and an enzyme known as the Sleeping Beauty transposase have been used to genetically modify a vertebrate animal. In the study, which is published in the April 2 issue of the Proceedings of the National Academy of Sciences, University of Minnesota researchers injected a transposon containing the gene for a yellow coat color into a mouse embryo, resulting in a genetically modified mouse.
"This is a new type of technology, an entirely different way to make genetically modified animals," said David Largaespada, Ph.D., an assistant professor in the university's department of genetics, cell biology and development and director of the University of Minnesota Cancer Center's Genetic Mechanisms of Cancer research program. "The Sleeping Beauty transposase enzyme plus the transposon is like a truck used to carry the cargo, or specific genes, into the animal. These specific genes could help treat diseases such as cancer."
Researchers at the Cancer Center injected a one-cell mouse embryo--a fertilized egg--with a linear piece of DNA containing the transposon, along with a source of Sleeping Beauty transposase enzyme. In this case, the transposon contained the gene for making a yellow-colored coat. The enzyme then caused the transposon to "jump" from the linear piece of DNA to a mouse chromosome, where it was able to express its function of coat color.
"We're very excited about Sleeping Beauty’s potential," said Largaespada. "One use would be to add genes to germ cells or early embryos in order to produce large amounts of a protein in an animal. The protein then would be purified and used as a drug treatment for hemophilia, for instance."
Another function would be to create genetically altered farm animals, which could be used as a source of organs for transplantation. Gene identification and function may also be determined by transposons' ability to mutate or "knock out" genes.
"We and other scientists at the university, such as Scott McIvor, are also working on transferring genes directly into cells of the body, in the liver or lungs, for instance," said Largaespada. "We hope this procedure could help cure diseases such as cystic fibrosis or hemophilia."
Largaespada is co-founder of Minneapolis-based Discovery Genomics, Inc., which has exclusive license to Sleeping Beauty and plans to commercialize this technology. http://www.discoverygenomics.net/
The University of Minnesota Cancer Center is a National Cancer Institute-designated Comprehensive Cancer Center. Awarded more than $68 million in peer-reviewed grants during fiscal year 2001, the center conducts cancer research that advances knowledge and enhances care. The center also sponsors community outreach and public education efforts addressing cancer. For more information on cancer in general, visit the Web site at http://www.cancer.umn.edu or call (1 888-CANCERMN, 1-888-226-2376 or 612-624-2620).
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