Scientists bypass major hurdle to hemophilia gene therapy

Scientists bypass major hurdle to hemophilia gene therapy; animal studies yield high levels of clotting protein

CHAPEL HILL - For the first time, scientists at the University of North Carolina at Chapel Hill have used a gene-therapy technique in animals to continually produce very high amounts of a clotting protein similar to that lacking in people with hemophilia.

A lack of this protein, known as factor IX, occurs in hemophilia type B. If human gene therapy studies could yield sustained factor IX production in high amounts, then hemophilia patients would no longer need daily injections of the protein. The new research suggests this might someday prove feasible.

Moreover, the new findings also indicate that the gene-therapy method used in the study may be applied to hemophilia A, the more common form of the disease. And a report published Dec. 4 in the journal Molecular Therapy concludes that the approach "may be useful for the treatment of a wide variety of inherited diseases."

In animal experiments at Carolina and elsewhere in recent years, the method involved a genetically engineered virus called AAV to infect cells and thereby deliver a cloned gene into an animal's body. Previous studies used only type 2 of six known AAV serotypes, each of which differ in their protein wrapper. This time, however, the Carolina researchers tried five of the six, comparing factor IX production of AAV types 1, 3, 4 and 5 with that of type 2. The results were startling.

Surprisingly and unexpectedly, we found the mice were producing amounts of this factor 100 to a thousand times more than weve observed before, said senior study author Dr. Christopher E. Walsh, assistant professor of medicine at the UNC-CH School of Medicine and clinical director of the universitys Gene Therapy Center.

"The purpose of this experiment was to see if we could generate very high protein production long-term," he added. "These mice are now six months out

Contact: Leslie Lang
University of North Carolina School of Medicine

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