Rett syndrome is a debilitating genetic disorder of the brain, resulting in very severe physical and cognitive disability. It is mostly caused by mutations (errors in the genetic code), in the “MECP2” gene, a gene that is vital for normal brain function.
Given the highly complex nature of the brain, developing treatments for genetic brain disorders such as Rett syndrome has always been challenging. While multiple studies have led to a better understanding of the disorder, none have yet led to a new treatment or cure for the disease.
The Molecular Neurobiology Research laboratory, headed by Dr Wendy Gold, is focussed on developing and applying advanced gene-editing technologies for the treatment of Rett syndrome.