Gene Therapy For Parkinson's Reported

Researchers at the University of Rochester in the USA have successfullyused a modified rhinovirus to deliver into rat brains the gene for glial cell-derived neurotrophic factor, which has been shown to protect neurons from degenerating, according to a report in Science. They also found that Parkinsonian rats treated with the injected therapy lived longer than those who were untreated.

Two months after the gene transfer, some of the rats were sacrificed and brain tissue was examined. In 12 rats who did not receive the treatment, 69% of neurons in the target area had been lost, while in the treated group (six rats) only 21% had been lost.

These results are similar to those achieved when GDNF was injected directly into the brain, a technique under investigation by researchers at Amgen, according to lead researcher Martha Bohn. However, the gene therapy approach, which results in the target neurons producing their own GDNF, requires fewer invasive injections, and means that the patient would be exposed to lower doses of GDNF.