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After Setbacks, Small Successes for Gene Therapy

Not long ago, gene therapy seemed troubled by insurmountable difficulties. After decades of hype and dashed hopes, many who once embraced the idea of correcting genetic disorders by giving people new genes all but gave up the idea

But scientists say gene therapy may be on the edge of a resurgence. There were three recent, though small, successes — one involving children with a fatal brain disease, one with an eye disease that causes blindness and one with children who have a disease that destroys the immune system.

“We are ready to move,” said Dr. Luigi Naldini of the Institute for Gene Therapy at Vita-Salute San Raffaele University in Milan.

Dr. Kenneth Cornetta, a gene therapy researcher at Indiana University and president of the American Society of Gene and Cell Therapy, added: “It’s exciting. The science gets better every year.”

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