GENE therapy is coming in from the cold. Two boys treated three years ago with a gene therapy for , the brain disease featured in the film Lorenzo’s oil, fared so well that doctors have treated a third and are now looking for adult volunteers.
“They have normal, family lives,” says of the Descartes University in Paris, France, a member of the team that pioneered the ALD gene therapy.
ALD is caused by a faulty gene that prompts the myelin sheath coating nerves in the brain to wear away, causing impaired speech, movement and eventually death.
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