At first blush, gene therapy seems ill-suited to treating Parkinson's disease (PD). Scientists have linked few cases to missing or mutated genes and are generally clueless about the disease's cause. But the need for some relief from its debilitating symptoms is so great that gene therapy researchers have labored over the past decade to develop counter-strategies. These studies have had promising results. When the brains of rat and monkey PD models express certain transgenes, the animals show less of the disease's hallmark pathology, a deterioration of nerve fibers that secrete the neurotransmitter dopamine. The animals also exhibit fewer behavioral abnormalities than do controls.

Armed with these findings, laboratory scientists and clinicians are now preparing to test gene therapy on humans with PD. Last spring, the National Institutes of Health Recombinant DNA Advisory Committee (RAC) reviewed a PD gene therapy protocol for the first time, endorsing various recommendations by a 12-0...


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