RNA Treatment Delivered Directly To Brain Can Help Treat Huntington's

Alnylam, a Cambridge, Massachusetts-based company, has announced a clinical trial for genetic therapy based on RNA interference, or RNAi whose single dose can have therapeutic effect on the target gene, lasting for over a month. The company is simultaneously working on a medical device maker, Medtronic, that will act as a carrier to deliver RNAi treatment directly to the brain, that can help in treating the degenerative brain disease Huntington's.

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The company claims that the highest dose of its RNAi, reduces the amount of faulty protein that triggers the disease by a staggering 94 percent. RNAi therapy is realized by producing snippets of RNA, similar to DNA in structure, that match a part of the target gene. This interfering RNA then destroys the gene's products before any protein is formed. Usually, the methods available today focus on binding to the protein that causes the disease, while RNAi stops the generation of protein in the first place.

The major hurdle that Researchers came across in supplying the therapeutic RNA was their short time of living in bloodstream. Hence, it was important that the small interfering RNAs (siRNA) were delivered to the correct cells. For Huntington's and diseases that are related to brain, the Researchers needed to get past the blood-brain barrier. To circumvent this, the device by Medtronic is implanted in patients which delivers liquids directly into the brain tissue.

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