Another small step forward in gene therapy (Parkinson's)

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Another small step forward in gene therapy (Parkinson's)

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It's a crude surgical procedure for delivery, and included only a few subjects with Parkinson's disease. They switched on synthesis of a specific protein in an area of the brain. As safety and methods of delivery improve, those with a predisposition to other age-related diseases might hope for effective treatments. Current limitations might not last forever. Even more worthwhile persisting with healthy living.

from http://ir.voyagertherapeutics.com/phoen ... ID=2337283

"VY-AADC, comprised of the adeno-associated virus-2 capsid and a cytomegalovirus promoter to drive AADC transgene expression, is designed to deliver the AADC gene directly into neurons of the putamen where dopamine receptors are located, bypassing the substantia nigra neurons and enabling the neurons of the putamen to express the AADC enzyme to convert levodopa into dopamine. The approach with VY-AADC, therefore, has the potential to durably enhance the conversion of levodopa to dopamine and provide clinically meaningful improvements by restoring motor function in patients and improving symptoms following a single administration."
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