Pharmaceutical Business review

Researchers make breakthrough in neurological disorder

Dr Adrian Bird and his colleagues have found that the “Rett Syndrome protein”, MeCP2, only binds to genes with a specific sequence of nucleotide bases. This knowledge will aid in the identification of the genes that are regulated by the gene MECP2, moving researchers a step closer to finding an effective treatment for the disease.

Rett syndrome (RTT) is a disorder diagnosed almost exclusively in girls. Children with RTT appear to develop normally until 6 to 18 months of age, when they enter a period of regression, losing speech and motor skills. Most develop repetitive hand movements, irregular breathing patterns, seizures and extreme motor control problems. RTT leaves its victims profoundly disabled, requiring maximum assistance with every aspect of daily living. There is currently no cure for the disease.

“Finding the MeCP2 target genes is a crucial step in understanding what goes awry in Rett Syndrome. Unfortunately these genes have been elusive. Dr Bird’s discovery… provides a much-needed clue which should aid in their identification,” said Monica Coenraads, Director of Research for the Rett Syndrome Research Foundation.