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In-gene-ious

How a new gene targeting technique provided Eric Hendrickson with $6 million in federal grants, a biotech contract and new ways to treat life-threatening conditions.

If you’re looking for an expert at using recombinant Adeno-Associated Virus (rAAV) to modify human somatic cells, head on over to Eric Hendrickson’s laboratory on the 6th floor of the Molecular and Cellular Biology Building.

Hendrickson, professor of biochemistry, molecular biology and biophysics, has become one of the world’s leading experts at using the technology since he learned about it eight years ago.

Adenoviruses are the pathogens that cause the common cold. Adeno-Associated Virus (AAV) is an infectious, but non-pathogenic, agent that hitches a ride with adenoviruses yet doesn’t trigger an immune response. Approximately 85 percent of Americans today are seropositive for AAV, with no obvious pathogenic consequences. Importantly, the recombinant form of AAV can be manipulated to target genes but it has a distinct advantage over other gene targeting vector strategies since the immune system doesn’t chase it off.

The original description of the rAAV technique was made in David Russell’s laboratory (University of Washington) in 1998. Many researchers dismissed the report as too good to be true. But then Bert Vogelstein, a world-class cancer researcher at John Hopkins University, demonstrated that rAAV could be used to target oncogenes. When Hendrickson read this follow-up report his interest in the technology was piqued and he decided to try it in his own laboratory.

“I was skeptical at first that a truly wimpy, somewhat bizarrely structured single-stranded DNA virus could target genes with high efficiency but I decided to try it,” he says. “It not only worked, but it spawned everything that has happened in my laboratory since then.”

“Everything” includes about $6 million in federal grants to use the family of genes he studies to understand the molecular mechanisms that underlie the rAAV technology and to apply it to treat serious conditions, such as immune deficiency disorders, DNA damage caused by radiation therapy and cancer predisposition syndromes. Besides the federal research contracts, the Hendrickson laboratory is also supported by a research contract with Horizon Discovery, Ltd.  Horizon is the company that owns the patents for using rAAV to target genes. The Hendrickson laboratory is working on methodologies to improve and expand the uses of rAAV.

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