Experimental Therapy Repairs Faulty Autism Gene

Exciting results in the lab could translate to hope for patients with autism

Microscopy images reveal significant differences in size and structure between brain organoids derived from a patient with Pitt-Hopkins Syndrome (right) and from a control (left). Image copyright: UC San Diego.

Scientists have successfully used a new gene therapy to repair a genetic defect associated with autism. University of California San Diego School of Medicine neuroscientists say that this breakthrough finding, published in Nature Communications, paves the way for a new dawn in autism therapies.