Brain Organoids Help Illuminate and Test Treatments for Rare Form of Epilepsy

Animal models don’t consistently recapitulate human CDKL5 deficiency disorder (CDD), a rare form of epilepsy. Using personalized brain organoids — “mini-brains” in a lab dish derived from stem cells of patients deficient in CDKL5 protein — researchers at UC San Diego School of Medicine were able to, for the first time, mimic the altered electrical activity characteristic of CDD, detail the molecular mechanisms that malfunction, and test potential therapies to correct them.