From genes to treatments

This week, researchers in the US discovered that a drug currently used to treat a rare genetic childhood condition can stop the development of Parkinson's in mice. The drug, phenylbutyrate, is now being tested in people with Parkinson's.

The drug works by turning on a gene, called DJ-1, which helps the dopamine-producing nerve cells usually lost in Parkinson's to survive.

But what's exciting about this breakthrough - aside from the promise of a new treatment that could slow or stop Parkinson's - is that the foundations were laid by the discovery of problems in the DJ-1 gene in people with Parkinson's back in 2003.

Now, understanding why the DJ-1 gene is important for the nerve cells lost in Parkinson's has led to this potentially neuroprotective treatment. We funded a lot of the early work around this and discovered 2 key genes that are involved in Parkinson's.

And there's more to come.

Researchers at the John Hopkins Institute for Cell Engineering in the US have unravelled how another crucial gene causes nerve cells to die in Parkinson's.

And just last month 5 new genes linked to Parkinson's were identified in the largest genetic study of the condition to date. This brings us up to 11 genes that we now believe play a part in Parkinson's.

We are now beginning to see the pieces of this complex puzzle of genes fitting together. And this is leading to the first tantalising glimpses of treatments that tackle the root causes of Parkinson's by targeting the genes involved in the condition.

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