Gene therapy breaking ground in treating Parkinson’s disease

|

Parkinson’s patients who take the drug levodopa, or L-Dopa, are inevitably disappointed…[when,] over time[,] the drug becomes less effective…

A biotech company called Voyager Therapeutics now thinks it can extend the effects of L-Dopa by using a surprising approach: gene therapy. The company…is testing the idea in Parkinson’s patients who’ve agreed to undergo brain surgery and an injection of new DNA.

Voyager’s strategy…is to inject viruses carrying the gene for AADC into the brain, an approach it thinks can “turn back the clock” so that L-Dopa starts working again in advanced Parkinson’s patients as it did in their honeymoon periods.

[There is] one edge [that] Voyager’s approach has over others. It is possible to tag AADC with a marker chemical, so doctors can actually see it working inside patients’ brains…[In] past studies of gene therapy, by contrast, doctors had to wait until patients died to find out whether the treatment had been delivered correctly.

“I believe that previous failure of gene-therapy trials in Parkinson’s was due to suboptimal delivery,” says [Krystof] Bankiewicz.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion and analysis. Read full, original post: Manufacturing Dopamine in the Brain with Gene Therapy