Thorsten Stafforst, PhD
University of Tübingen
Basic Research Award: $149,600
Harnessing RNA editing to treat Rett syndrome
Gene-modifying therapies are thought to possess great potential for treating genetic disorders such as Rett syndrome because they target the genes that cause disease. Dr. Staffort’s lab has developed a new approach to gene-modifying therapy that harnesses the RNA editing machinery already present inside cells. This approach works by directing the RNA editing machinery to repair the RNA intermediate made from a mutant gene so that a normally functioning protein is made instead of the mutant one that causes disease. It is advantageous in that the changes that it makes are not permanent and their dosage can be controlled. This research will test for the first time if this approach can treat diseases such as Rett syndrome that are caused by a single gene mutation using mouse models.