A science powerhouse bets on genetic therapy to beat brain disorders
Scientists who've spent decades learning how the brain works say they're now ready to start fixing it when it breaks.That's the premise of the Brain Health accelerator, a collaborative effort launched by the Allen Institute in Seattle, which has become a major player in brain research.The initiative includes plans to develop new genetic therapies — a term that includes gene editing as well as traditional gene therapy — for diseases including Alzheimer's, Parkinson's, ALS, and Huntington's."The latest genetic treatments allow scientists to control the activity of particular genes," says Ed Lein, who directs the institute's brain health programs."That opens up the possibility for very specific precision therapies for brain disorders."The accelerator is an outgrowth of the BRAIN Initiative, an ambitious research program unveiled by President Obama in 2013.
The goal of this public-private partnership was to create tools that would allow scientists to see the brain's inner workings, and, eventually, to develop treatments.But the effort has progressed far faster than many scientists expected."I am shocked at how far we've come in the last 10, 12 years," says John Ngai, a senior investigator at the National Institutes of Health who directs the BRAIN Initiative."It's just been beyond my wildest imagination — and I've been accused of having a pretty good imagination."The Allen Institute's Brain Health accelerator is attracting a range of scientists intent on translating their knowledge about the brain into treatments for its disorders.Jeff Carroll, for example, was a teenager when he learned that his mother had Huntington's Disease, a fatal, inherited disorder that destroys brain cells."The whole reason I'm in science started with this frustration with not being able to understand what was happening with my mom," he says.Carroll eventually learned that he, too, carried the Huntington's gene.He spent years at the University of Washington studying mice with the conditi...