February 17, 2009

Gene therapy may fight cystic fibrosis

U.S. scientists have turned a relatively benign virus into an infectious form that, in its first gene therapy test, cured cystic fibrosis tissue in culture.

Researchers from the University of California-Berkeley and the University of Iowa said their achievement with the benign adeno-associated virus overcomes a major problem of earlier virus-based gene therapy for cystic fibrosis, setting the stage for tests in advanced animal models of the disease.

I think it is worthwhile thinking about clinical therapy at the levels of infection we are achieving, said UC-Berkeley Professor David Schaffer.

He said a new pig model of cystic fibrosis developed last year by pulmonologist Professor Joseph Zabner at the University of Iowa will provide a key test of the virus as a carrier of a gene to replace the mutated gene responsible for the disease.

If we are able to show that efficient gene transfer can result in gene therapy, if we can cure the lung disease of pigs that have been genetically engineered to have cystic fibrosis lung disease, we should have a real chance of curing cystic fibrosis in humans, Zabner said.

The research appears in the online early edition of the Proceedings of the National Academy of Sciences.