‘Safety-Catch’ May Have Been Found In Gene Therapy For Parkinson’s

August 29, 2009

Florida (ChattahBox) – Scientists have found that the application of a certain antibiotic shuts off a gene therapy that is being developed in the fight against Parkinson’s.

The finding shows a specific ability to stop gene therapy if problems begin to occur, specifically during the experimental phase. It offers a kind of ‘safety-catch’, which provides added security as researchers look for a way to dismantle the disease.

In addition, scientists have found that the gene therapies being used may have been unsuccessful in the past specifically because it was delivered too late in the disease. They hope that introducing the therapy sooner will have more positive effects.

UF’s McKnight Brain Institute and the Powell Gene Therapy Center professor, Ronald Mandel, was quoted by EurekAlert:

“We have worked every day for 10 years to design a construct to the gene delivery vector that enhances the safety profile of gene transfer for Parkinson’s disease. With that added measure of safety, we believe we can intervene with gene transfer in patients at earlier stages of the disease.

“We strongly believe that trials to save dopamine-producing connections in patients with Parkinson’s disease have failed because the therapy went into patients who were in the late stages of the disease and who had too few remaining dopamine-producing connections.”

Hopefully, doctors will be able to use this antibiotic side effect to regulate the effects of the gene therapy after it is delivered.

The study will be published in September’s online edition of Molecular Therapy.


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