Scientists halt brain disease with gene therapy

Deactivated version of AIDS virus stalls rare and fatal ailment in two boys

Scientists have managed to halt a rare and fatal brain disease with an experimental gene therapy technique using a deactivated version of the AIDS virus, a study published on Thursday showed.

The international team used a disabled form of human immunodeficiency virus (HIV) to deliver working genes to two boys with the brain disease X-linked adrenoleukodystrophy (ALD). Their success may help shape future treatment.

Patrick Aubourg of Inserm-University Paris Descartes, who led the study, said it was the first time scientists had successfully used an HIV-derived delivery technique for gene therapy in humans, and the first time gene therapy had been used effectively in a severe brain disease.