In a First, Gene Therapy Halts a Fatal Brain Disease

For the first time, doctors have used gene therapy to stave off a fatal degenerative brain disease, an achievement that some experts had thought impossible. The key to making the therapy work? One of medicine’s greatest villains: HIV. The patients were children who had inherited a mutated gene causing a rare disorder, adrenoleukodystrophy, or ALD. Nerve cells in the brain die, and in a few short years, children lose the ability to walk or talk.

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