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Jennifer Adair carried out her first gene therapy experiment several years ago. A cancer treatment, it involved collecting blood from a patient, and then adding to these cells a new strand of DNA—a gene—that would protect them from a powerful chemotherapy. The altered cells were then reinfused into their veins.

The study, involving 11 patients, proved fairly successful. But Adair, who runs a gene-therapy lab at the Fred Hutchinson Cancer Research Center in Seattle, says it took three sleepless scientists around 96 hours just to process a single person’s cells in a multimillion-dollar clean room. “I said, ‘Wow, we really need to simplify this,’ ” Adair recalls.

Gene therapy is moving quickly from experiment to medical reality. But with potential treatments for cancer and rare diseases now showing promise, scientists are worried that the technology is so complex that patients will not benefit as quickly as they should because of a shortage of trained technicians and suitable facilities. For the most successful gene therapies, those that require modifying blood cells outside the body, the procedures are offered only by a dozen or so research centers, all in major cities like New York, Seattle, Milan, and Paris.

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MIT Technology Review

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