Testing a Genetic "Ad-Blocker" to Treat Disease

Researchers at the University of Chicago are using a novel approach to try to halt a rare neurological disorder: a sort of genetic "ad-blocker" that appears to be effective — at least in baby mice.

The disorder, Spinocerebellar ataxia type 6, or SCA6 for short, is a rare neurological disease similar to ALS (Lou Gehrig's Disease) or Parkinson’s. Researchers identified the gene that codes for the disease-causing protein in SCA6, but there was one problem. The gene that controlled the disease-causing protein also coded for a second protein that is essential for life.

Think of it this way: You click on a link to watch a movie, but as soon as you click on it, the movie opens but so does a virus that destroys your computer. How do you watch the movie without breaking your computer? Downloading an ad-blocker would do the trick. The researchers needed something similar.

 

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