In the early 2000s, gene therapy seemed to be on life support. The once-promising technique, which uses engineered viruses and other methods to shuttle genes into human cells to fix DNA errors, faced a setback after an 18-year-old man died during a clinical trial in 1999. Later analysis showed that the virus carrying the DNA fixes had triggered a massive immune reaction that caused the man’s organs to shut down.
But after years of troubled times, the field is on the move again. Success stories, an infusion of venture capital and regulatory approval in the European Union have revitalized the field and made scientists experienced in the technology hot commodities once more. “The field has really started to take off again,” says Harry Gruber, chief executive of Tocagen, a gene-therapy company based in San Diego, California. The number of people working in gene therapy has grown enormously, he says, and that is going to continue.
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