BRONTE COOREY

Rett syndrome is a rare neurodevelopmental disorder which is highly complicated and regulated by the expression pattern of the Methyl-CpG-binding Protein 2 (MECP2) gene. My projects looks at editing the MECP2 gene at the native locus to correct pathogenic variants in the gene and restore functional activity of MECP2. I am testing this approach in vitro in patient cell lines, including fibroblasts and induced pluripotent stem cell (iPSC) derived cortical brain organoids, as well as in vivo in the T158A Rett syndrome mouse model.