In 2003, a team of researchers working for the Department of Energy and the National Institutes of Health completed a 13-year long effort called the Human Genome Project. The goal of the project was, among other things, to identify and map every gene in human DNA. For a while, the scientific community has been looking at the data and wondering, "So, what's next?"
One answer is targeted therapy, medical treatments that do not only address symptoms, but also respond to a patient's genetic makeup.
The past couple of years have yielded breakthroughs in this field. In 2011, a team of scientists at the University of Pennsylvania effectively treated leukemia patients by engineering their own white blood cells to fight the disease. Also in 2011, researchers in Britain achieved promising results in treating the genetic disease hemophilia B by injecting patients with a re-engineered version of the gene. 2012 brought even more advancements, using targeted therapy to treat inherited blindness.
"Talk about transforming an industry," says George Day, co-director of the Mack Center at Wharton. "Big Pharma has always been pill-based," he says, meaning patients need daily dosage, whereas "gene therapy is one and done.
"I think finally after 20 years, the promise of gene therapy is starting to be realized."
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