Despite a roller-coaster ride of ups and downs during the past 15 years, gene therapy has continued to attract many of the worlds 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.
Dian Land | EurekAlert!
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