Gene Therapy Halts Brain Disease in Two Boys

Posted on: November 8, 2009

Researchers have used a modified AIDS virus to halt a devastating brain disease in two young boys. The treatment, in which the virus delivered a therapeutic gene, marks the first time gene therapy has been successfully used against X-linked adrenoleukodystrophy (ALD)–a disorder that is always fatal if untreated. With this proof of principle, scientists hope versions of the AIDS virus engineered to carry different genes can now be applied to a variety of other diseases.

via Gene Therapy Halts Brain Disease in Two Boys — Kaiser 2009 (1105): 1 — ScienceNOW.

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1 Response to "Gene Therapy Halts Brain Disease in Two Boys"

The use of viruses to deliver gene therapy or to deliver drugs is a very promising area of disease treatment. As the approaches mature, it will be interesting to see whether nanoparticles or viruses become the predominant means for accomplishing such tasks.

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