February 17, 2009
Professor of Bioengineering and Chemical Engineering David Schaffer, along with researchers from UC Berkeley and the University of Iowa, have turned a relatively benign virus into a highly infectious form that is ideal as a carrier for gene therapy.
In its first test, it completely cured human cystic fibrosis lung tissue in culture. UC Berkeley researchers forced the adeno-associated virus to evolve so as to bind to sialic acid on the surface of lung cells, making it easier for the virus to infect them.
This success with the benign adeno-associated virus (AAV), published this week in the online early edition of Proceedings of the National Academy of Sciences, overcomes a major problem of earlier virus-based gene therapy for cystic fibrosis, and sets the stage for tests in advanced animal models of the disease.