Rett syndrome: new brain organoid developed for testing therapies

A team of researchers from the University of California San Diego School of Medicine (CA, USA) have collaborated with StemoniX Inc. (MN, USA) to test a range of drugs in a brain organoid model of Rett syndrome. Nefiracetam and PHA 543613 were found to resolve molecular and cellular symptoms in the organoids. The authors of the paper, published in EMBO Molecular Medicine, hope that these two drugs can be tested in clinical trials in patients with Rett syndrome. While most autism spectrum disorders are associated with a range of complex genetic factors, Rett syndrome is categorized by mutations in a...