Tinkered Virus 'Cured' Cystic Fibrosis in Lab

Researchers from the University of California, 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 gene therapy test, it completely cured human cystic fibrosis lung tissue in culture.

This success with the benign adeno-associated virus (AAV), published this week in the online early edition of the journal 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.

"I think it is worthwhile thinking about clinical therapy at the levels of infection we are achieving," said coauthor David Schaffer, professor of chemical engineering at UC Berkeley.

A new pig model of cystic fibrosis developed last year by Schaffer's colleague, pulmonologist Joseph Zabner of the University of Iowa Hospitals and Clinics in Iowa City, 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 in an e-mail.

Schaffer's lab is collaborating with groups elsewhere to adapt the virus to gene therapy for other diseases, including Alzheimer's disease and amyotrophic lateral sclerosis (Lou Gehrig's disease).

"Both of those are situations where improvements in the properties of the vehicle can have a significant impact on the success of the therapy,"
Schaffer said.

I've always felt that gene therapy was being undersold while stem cells have been waaaaaaaaaaaay oversold. There is SOOOO much potential for, well, cures from lots of nasty diseases that are genetic in nature that stem cells are only patching...at best.

Besides, stem cells can and do cause cancer too. GT also has less squicky bits morally, too, fora lot of people. Oh wait, we're still messing with God's work. Doh.

That's just me. More money for gene therapy!