Most autism spectrum disorders have a complex, multifactorial genetic component, making it difficult to find specific treatments. Rett syndrome is an exception. Babies born with this form of the disorder have mutations specifically in the MECP2 gene, causing a severe impairment in brain development that primarily affects females. Yet there is still no treatment — current therapies are aimed at alleviating symptoms, but don’t address the root cause.
Researchers at University of California San Diego School of Medicine and Sanford Consortium for Regenerative Medicine recently used stem cell-derived brain organoids — also called “mini-brains” — that lack the functional MECP2 gene to better study the disease.
