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Scientists Turn Benign Virus into Effective Gene Therapy Carrier for Cystic Fibrosis
The researchers have revealed that the first gene therapy has been found to completely cure human cystic fibrosis lung tissue in culture.
Writing about their achievement in the online early edition of the journal Proceedings of the National Academy of Sciences, the researchers said that their success with the benign adeno-associated virus (AAV) 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 co-author David Schaffer, professor of chemical engineering at UC Berkeley.
Schaffer's pulmonologist colleague Joseph Zabner, who developed a new pig model of cystic fibrosis last year at the University of Iowa Hospitals and Clinics in Iowa City, has revealed that this model 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.
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More News on: Chicken Pox, DNA Finger Printing, Shigellosis, Reiki and Pranic Healing, Genetic Testing of Diseases, Pancreas, Cystic Fibrosis, Niemann-Pick Disease
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