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The Gene Hunters

 
. .

Hope for Gene Therapy

4 pages: | 1 | 2 | 3 | 4 |

Bumps in the Road

"This has to be allowed to be rigorously tested," insists Isner. "Cooler heads will prevail."

But Insner has little more than "hints" to go on. In 1989, after he injected the first genetically engineered human cells into the first human patient, W. Anderson French told the Washington Post, "We were ready for every problem except the patient dying. That would have set the whole field back."

Ten years later, Anderson's nightmare scenario played itself out when a patient enrolled in a University of Pennsylvania gene therapy trial died. Eighteen-year-old Jesse Gelsinger suffered from a liver disorder that allowed poisonous levels of ammonia to build up in his body. His death on September 17th, 1999 prompted the FDA to investigate and eventually halt all human genetic experiments by the Penn researcher in charge of Gelsinger's trial.

A few months later, following the death of two of his 45 patients and conflict-of-interest allegations, the FDA shut down Inser's work as well. Inser maintained the patients died from the end-stage heart disease that brought them to the study in the first place. Despite not being able to collect data from those trials, Isner saw some promising results, promising enough for him to fight for the right to continue his trial. "This has to be allowed to be rigorously tested," he insists. "Over the last year, there has been a lot of concern about the field of gene therapy in the wake of what was often inaccurate reporting in the media. But, cooler heads will prevail. Gene therapy is getting a second look right now."

Photo of woman at mall
  Today, Isner patient Lillian Cooper is going strong, but the future of the gene therapy remains uncertain.

Studies similar to Isner's work with the VEGF gene are currently underway at Cornell University and in Europe. Isner's optimistic that this research will ultimately lead to the gene therapy holy grail- learning how to mend the genetic typos that cause heritable disease. "While our form of gene therapy does not directly teach us about how to treat inherited disorders," Isner forecasts, "it makes sense that clinical experience with it will give us tips and hints about how to do that one day."
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4 pages: | 1 | 2 | 3 | 4 |

Scientific American Frontiers is sad to report the death of Dr. Jeffrey Isner. More on his life's work

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