The Molecular Neurobiology group focuses on improving our understanding of complex neurodevelopmental disorders including Rett syndrome, autism spectrum disorders, Tourette syndrome and obsessive-compulsive disorder.
The group’s main focus lies in the development of gene therapies to cure Rett syndrome, a neurodevelopmental condition which almost exclusively affects girls. This devasting disorder leads to a loss of motor skills and speech, where symptoms present from around 6 months of age. Girls suffering from Rett syndrome also experience repetitive hand movements, seizures, scoliosis and breathing problems. Rett syndrome affects 1 in 10,000 live female births, with the main cause involving mutations in the MECP2 gene. This gene codes for a finely controlled protein, which is essential for regulating correct gene expression and brain development.
Gene therapy is an emerging therapeutic approach, which involves repairing the expression of a gene to treat or cure diseases. The Molecular Neurobiology is led by A/Prof Wendy Gold and includes a number of key people.