Nancy LUO

Rett syndrome is a rare neurodevelopmental disorder, affecting 1 in 10 000 children. It is characterised by mutations within the X-linked Methyl-CpG-binding Protein 2 (MECP2) gene that encodes for the MeCP2 protein. This project aims to develop a gene replacement therapy to restore physiological levels of functional MeCP2 expression. To test the gene therapy, MeCP2 expression in vitro cell cultures and behavioural tests of in vivo Rett mouse models will be used to  determine and fine-tune treatment efficacy.