Major Advance Made in Gene Therapy
Researchers find safer way to deliver therapeutic DNA to cells
HealthDayNews — A major advance in solving a stumbling block of gene therapy — how to safely and effectively deliver therapeutic DNA inside cells — has been reported University of Wisconsin scientists.
In what’s described as a remarkably simple solution, they used a system similar to that used for intravenous injection (IV) to inject genes and proteins into the limb veins of laboratory animals. The injected genetic material made its way to the animals’ muscle cells and functioned properly for an extended period.
“I think this is going to change everything relating to gene therapy for muscle problems and other disorders,” gene expert Jon Wolff said in a prepared statement.
“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 said.
Many other scientists have experimented with using harmless viruses to deliver DNA to cells.
The UW research was presented June 3 at the annual meeting of the American Society for Gene Therapy.
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