A science powerhouse bets on genetic therapy to beat brain disorders

After spending two decades studying how the brain works, a team of scientists prepares to focus on gene therapy treatments for brain diseases, including Alzheimer's and Parkinson's. NPR Science LISTEN & FOLLOW Science A science powerhouse bets on genetic therapy to beat brain disorders June 3, 20264:43 PM ET Heard on All Things Considered Jon Hamilton A science powerhouse bets on genetic therapy to beat brain disorders Listen · Transcript Toggle more options Download Embed Embed <iframe src="https://www. npr. org/player/embed/nx-s1-5837620-e1/nx-s1-9796299" width="100%" height="290" frameborder="0" scrolling="no" title="NPR embedded audio player"> Transcript After spending two decades studying how the brain works, a team of scientists prepares to focus on gene therapy treatments for brain diseases, including Alzheimer's and Parkinson's. AILSA CHANG, HOST: Scientists who have spent decades learning how the brain works say they are ready to start fixing it when it breaks. A brain research center in Seattle called the Allen Institute is working to develop genetic therapies for diseases including Alzheimer's, Parkinson's, ALS and Huntington's. JON HAMILTON, BYLINE: The effort is called the Brain Health accelerator, and it includes scientists like Jeff Carroll. When Carroll was a teenager, he found out his mother had Huntington's disease. It's a fatal inherited disorder that destroys brain cells. JEFF CARROLL: The whole reason I'm in science started with this frustration with not being able to understand what was happening with my mom. HAMILTON: Carroll learned that he, too, carried the Huntington's gene. He spent years at the University of Washington studying mice with the condition, which occurs when the faulty gene tells nerve cells to produce toxic levels of a certain protein. To Carroll, the solution seemed obvious. CARROLL: Since we know all the bad stuff in Huntington's comes from this one gene, let's get rid of the levels of that gene. HAMILTON: But finding a way to do that was beyond the reach of his own lab. So Carroll joined the Allen Institute. CARROLL: It's difficult to do the scale of research that you need with a team of five or six or even 10 people, and so the hundreds of people they have here at the Allen Institute is just an entirely different approach to science. HAMILTON: The scientist in charge of the Brain Health accelerator is Ed Lein. He says it's built on years of basic research. ED LEIN: We now have a complete description of the types of cells that make up the brain and also the genetic underpinnings of their properties.