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Researchers report major advance in gene therapy technique

MADISON - Despite a roller-coaster ride of ups and downs during the past 15 years, gene therapy has continued to attract many of the world's brightest scientists. They are tantalized by the enormous potential that replacing missing genes or disabling defective ones offers for curing diseases of many kinds.

One group, consisting of researchers from the University of Wisconsin Medical School, the Waisman Center at UW-Madison and Mirus Bio Corporation of Madison, Wis., now reports a critical advance relating to one of the most fundamental and challenging problems of gene therapy: how to safely and effectively get therapeutic DNA inside cells.

The Wisconsin scientists have discovered a remarkably simple solution. They used a system that is virtually the same as administering an IV (intravenous injection) to inject genes and proteins into the limb veins of laboratory animals of varying sizes. The genetic material easily found its way to muscle cells, where it functioned as it should for an extended period of time.

"I think this is going to change everything relating to gene therapy for muscle problems and other disorders," says Jon Wolff, a gene therapy expert who is a UW Medical School pediatrics and medical genetics professor based 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."

Wolff conducted the research with colleagues at Mirus, a biotechnology company he created to investigate the gene delivery problem. He will be describing the work on June 3 at the annual meeting of the American Society for Gene Therapy in Minneapolis, and a report will appear in a coming issue of Molecular Therapy. The research has exciting near-term implications for muscle and blood vessel disorders in particular.

Duchenne's muscular dystrophy, for example, is a genetic dise
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Contact: Dian Land
dj.land@hosp.wisc.edu
608-263-9893
University of Wisconsin-Madison
3-Jun-2004


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