Stem Cell-Derived ‘Mini-Brains’ Reveal Potential Drug Treatment for Rare Disorder

September 10, 2015

(Medical Xpress) – Using “mini-brains” built with induced pluripotent stem cells derived from patients with a rare, but devastating, neurological disorder, researchers at University of California, San Diego School of Medicine say they have identified a drug candidate that appears to “rescue” dysfunctional cells by suppressing a critical genetic alteration. Their findings are published in the September 8 online issue of Molecular Psychiatry.