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Gene Therapy May Protect Against Flu Pandemics

Study found coaxing cells in the nose to make super antibodies protected mice and ferrets from pandemic strains

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In the test, treated and untreated mice and ferrets were exposed to several different flu strains that have caused worldwide epidemics, including the notorious H1N1 strain that caused the 1918 pandemic, which killed somewhere between 30 million and 50 million people.

"The amount of virus we gave them is 100-fold more than would normally kill them if they weren't treated," Wilson said.

Untreated animals quickly succumbed to the virus, but most animals treated with the gene therapy had some protection against the flu strains they were exposed to. Between 50 percent and 100 percent of the animals survived.

Importantly, 100 percent of the treated animals survived the H1N1 strains that caused the 2009 and 1918 pandemics.

"It really shows that the antibody, when delivered the right way, really has the ability to block infection and prevent disease," said Wilson, who said he has a financial stake in the technology used to deliver the gene therapy.

The study was published in the May 29 issue of the journal Science Translational Medicine.

Other researchers praised the study, but said many questions still need to be answered before it can be used safely in humans.

"It is promising," said Dr. Dimitrios Moskofidis, a virologist and immunologist at Georgia Regents University in Augusta. "The question is, how long could this protection last?" he said, and whether it's safe to coax non-immune cells into making immune proteins.

Study author Wilson said the next step to try to answer those questions involves human pilot studies, in which people would be given the gene therapy treatment and then exposed to weakened flu viruses to see if they get sick.

If it works with the flu, Wilson said, it might work for other respiratory diseases.

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