Gates-inspired gene therapy gives pandemic protection

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Vaccine pioneer Louis Pasteur is quoted as saying "chance favors the prepared mind," and the history of science is filled with seemingly random events that led to breakthroughs. Now it might have another. A meeting with Bill Gates has inspired a pandemic flu gene therapy.

Gates met University of Pennsylvania gene therapy researcher James Wilson in 2010. At the time Wilson was investigating the use of adeno-associated viruses (AAVs) as a gene-delivery vehicle to treat inherited diseases like cystic fibrosis and hemophilia. Gates questioned whether AAVs could be used to protect against a pandemic or emerging infection. Three years later Wilson has a paper in Science Translational Medicine answering Gates' question.

Wilson and other researchers from Pennsylvania and Canada used a spray to deliver genes into the nasal lining of mice and ferrets. The virus turned the animals' epithelial cells into mini antibody factories, which protected them against a range of pandemic viruses, including the H1N1 strain that caused the 1918 outbreak. Technically the therapy is not a vaccine--as it engineers cells to produce antibodies instead of stimulating an immune response--but it meets some of the goals of a universal flu shot.

Having a single nasal spray protect against a range of flu viruses would reshape how countries defend themselves against pandemics. National Institute of Allergy and Infectious Diseases Director Anthony Fauci described the work as an "important proof of concept." Talking to the Wall Street Journal, Fauci--who was not involved with the research--said: "You would already have in your hand something you can administer to people, rather than having to isolate the virus and starting making a vaccine."

The big limitation is the duration of protection, which waned after three months. This is too short a for a pandemic, so the team is trying to stretch the period of efficacy to 6 months. The goal is not to produce antibodies forever though, Wilson told ScienceNOW, in part because of safety concerns. Wilson is a founder of ReGenX Biosciences, which holds a patent related to AAVs.

- here's the WSJ article (sub. req.)
- check out the abstract
- get more in the ScienceNOW blog
- read the AFP's take

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