One Couple’s Tireless Crusade to Stop a Genetic Killer
Through mutual friends, the couple had met a scientist named (WWU Professor of Psychology) Jeff Carroll, who, like Vallabh, researches his own disorder—in his case, Huntington’s disease. He had recently partnered with a company called Ionis Pharmaceuticals to develop a therapy. Both fatal familial insomnia and Huntington’s result from a mutant protein that is toxic to brain cells. So how do you eliminate the protein? The simplest answer, Carroll explained, was to cut out the middleman. If DNA contains the architectural blueprint for a protein, a molecule called RNA is the contractor; it reads the schematics and specifies how the protein should be assembled. If you can intercept the RNA before construction has begun, you can affect the final shape of the building.