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Scientists develop method to introduce DNA into cells

Physician Practice Advisor, June 8, 2004

For 15 years scientist have puzzled over a fundamental question in gene therapy: How do you replace missing genes? Now, researchers from the Medical School, the Waisman Center, and Mirus Bio Corporation in Wisconsin have successfully inserted therapeutic DNA inside cells.

By injecting DNA without a protein coat (or "naked" DNA) directly into the veins of lab animals, the material found its way to the muscle cells without causing antibody responses or genetic reactions. Once injected, the introduced material remained in muscle cells for at least six months. This technique could be adapted to treat Duchenne's muscular dystrophy, arterial occlusive disease, and liver diseases, according to the report. 

"This is going to change everything relating to gene therapy for muscle problems and other disorders," said John Wolff, a medical genetics professor at the Waisman Center. "Our non-viral, vein method is a clinically viable procedure that lets us safely, effectively, and repeatedly deliver DNA to muscle cells. We hope that the next step will be a clinical trial in humans.

For more information, click here. http://www.news.wisc.edu/9867.html

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