Gene Target Beats Oil RemedyMarch 12, 2010, 9:00 am by Scientific American: Mind and BrainThe 1992 tearjerker Lorenzo’s Oil told the true story of one family’s struggle to save their son from X-linked adrenoleukodystrophy (ALD), a deadly degenerative brain disease. Unfortunately, over the ensuing years, the oil of the film’s title, a dietary supplement, has not panned out as the cure many people hoped it would be. Now a paper in the November 2009 issue of Science suggests that the long-sought cure may come from gene therapy--a famously hyped approach to treatment that tragically caused the death of a teenage experimental subject in 1999.Since then, however, researchers have continued to cautiously pursue gene therapy for certain disorders with known genetic origins. ALD, for instance, is caused by mutations in a gene called ABCD1, leading to unusually high levels of a type of fatty acid that damages the material insulating some neurons. It affects about one in 20,000 six- to eight-year-old boys, leading to death before adolescence. The main treatment is still bone marrow transplantation: a risky procedure that relies on finding a suitable donor, explains Patrick Aubourg, a neurologist at France’s INSERM research institute. [More]
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