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Cold, Flu, & Cough Health Center

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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


"The way I envisioned it was sort of a bioshield," Wilson said. "I wanted to focus the production of the antibody to the site where flu enters our body."

In animal tests, researchers showed that mice, ferrets and monkeys made many copies of the super antibody after they inhaled the gene therapy treatment. And the protection seemed to last for a while. Experts note, however, that promising research in animals often does not pan out in humans.

"In mice, it persists up to a year," Wilson said. "In monkeys, we think we're going to see expression up to six months."

The treatment also appears to work pretty quickly. Wilson said the animals were fully protected about three days after their nasal passages were swabbed.

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.

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