Nonviral Gene Delivery Forges Ahead

Jon Wolff, an MDA grantee at the University of Wisconsin-Madison, recently announced with that institution and the Madison biotech company Mirus that the group now has a European patent for a method of delivering "naked" genes (not encased in a viral shell) to muscle tissue via the bloodstream. The group already has a patent for this process in the United States.

Wolff, who has been working since the early 1990s on methods to deliver genes without using viruses, says his approach to intravenous delivery of "naked" DNA can put genes into a high percentage of muscle cells.

"This has prompted our desire to move it into the clinic," he said. "One of the concerns with the gene transfer technique has been its safety, since it requires high intravascular [inside blood vessels] pressure in order to be effective. However, we have conducted an extensive number of safety studies, and the technique appears to be very safe."

Most gene therapy techniques use viral casings to transport the therapeutic genes. The viruses are altered and are thought to be safe, but the possibility of an unwanted immune response to anything that looks like a virus remains a concern.

Mirus' lead gene therapy compound for muscular dystrophy is being developed with Transgene of Strasbourg, France, and the announcement says a clinical trial is planned for 2008.

It also says the delivery technique has potential for treating amyotrophic lateral sclerosis.