Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s disease.

In a study reported by Uniqure, which developed the experimental gene therapy, scientists found that it slowed progression of Huntington’s disease by 75% over three years. The study has not yet been published in a scientific journal.

“I went into the trial cautiously optimistic but very anxious, as one does when starting a gene-therapy trial,” says Dr. Sarah Tabrizi, director of the University College London Huntington’s Disease Center and a lead investigator on the study. “I was blown away when I saw all of the data and it was very, very clear that the gene therapy worked.”

The s

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