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In a first, gene therapy halts a fatal brain disease

The key to making the therapy work? One of medicine's greatest villains: HIV

Gina Kolata | NYT 

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. They become unable to eat without a feeding tube, to see, hear or think. They usually die within five years of ...

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First Published: Sat, October 07 2017. 22:18 IST