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Testing of gene therapies expanding at Boston-area hospitals

Kirsti Pigney is participating in a trial for gene therapy to treat blindness. She can’t tell whether the therapy is working, but said her vision hasn’t worsened.
Kirsti Pigney is participating in a trial for gene therapy to treat blindness. She can’t tell whether the therapy is working, but said her vision hasn’t worsened.Credit: Aram Boghosian for The Boston Globe

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Years after it was hyped, vilified for the death of a teenager, and then mostly forgotten by the general public, gene therapy has rebounded, and hospitals, companies, and investors in the Boston area have jumped on the bandwagon.

Patients are enrolling in a growing number of clinical trials here, and in some cases showing dramatic improvement. The technique, in which doctors “infect” patient cells with viruses engineered to carry useful genes, has matured and evolved.

Once trumpeted as a possible panacea for diseases ranging from sickle cell anemia to cystic fibrosis, gene therapy faced serious questions after the high-profile death of a Pennsylvania teenager in a clinical trial in 1999. But some researchers continued to work to overcome the safety and technical hurdles.

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