How the gene behind Huntington鈥檚 disease could be neutralized
Her most important slide showed decreases in the level of mutant huntingtin in trial participants鈥 CSF 鈥 indicative of reduced levels of the toxic protein in their brains 鈥 that were proportional to the amounts of drug the volunteers had received. At the two highest doses, production of the protein had, on average, decreased by about 40%.
鈥淧eople started crying,鈥 says Jeff Carroll, a neuroscientist who investigates Huntington鈥檚 disease at Western Washington University in Bellingham. 鈥淓verybody who works in Huntington鈥檚 disease long enough meets families and gets to know them, so it becomes very personal.鈥
Carroll鈥檚 connection to his work runs particularly deep. He began his career in neuroscience after his mother was diagnosed with Huntington鈥檚 disease. Then, in 2003, he discovered that he, too, had the mutation for the condition. Looking at Tabrizi鈥檚 slide, Carroll thought, 鈥淭his is a graph that is changing my life.鈥