January 30, 2004

Viral Gene Transfer Not Only Way,
MDA Researcher Says

Gene therapy — the insertion of functional versions of genes (DNA) to treat disease — often relies on the use of viruses to carry genes into the targeted cells.

While MDA continues to provide extensive support for viral delivery of therapeutic genes to treat genetic disorders, the Association isn’t ignoring other approaches.

MDA grantee Jon Wolff of the Department of Pediatrics at the University of Wisconsin at Madison says there are alternatives to viral transfer of DNA that may be safer and just as effective.

This week on WGN in Chicago, Wolff described his plans for using his “naked” (not encased in a virus) DNA transfer technique to get needed genes into the muscle cells of boys with Duchenne muscular dystrophy (DMD).

The gene that’s needed in DMD is one that leads to production of dystrophin, a protein that keeps muscle cells from tearing as they contract and relax.

In June, the French muscle disease association (Association Francaise Contre les Myopathies) announced that its grantees had safely injected naked dystrophin genes into the arm muscles of 15 boys with DMD or the related Becker MD (BMD). (See “French Gene Transfer.")

In doing so, the French group used technology developed in Wolff’s MDA-supported laboratory in Wisconsin.

Now, Wolff says, he wants to take his approach to the next level — using it in a human trial to deliver dystrophin genes to whole limbs by injecting them under pressure into the blood vessels instead of directly into muscles.

Wolff hopes to convince regulatory agencies of the soundness of his methods, which include putting each gene into a ring of supporting DNA (a plasmid) before injecting it into the body, then using a blood pressure cuff as a kind of tourniquet to increase injection pressure.

“The animal experiments look pretty promising,” Wolff says. “The human trials are in the planning stage.”