Small-molecule therapeutic boosts spatial memory and motor function in Rett syndrome mice
Girls born with a mutation in the MECP2 gene initially develop typically , but at 6-18 months a gradual or sudden reversal of development indicates the onset of Rett syndrome. As this rare genetic neurological disease develops, affected girls experience slowed growth, a loss of communication skills and use of the hands, problems with movement and coordination, difficulty breathing, and seizures. The disease almost exclusively presents in girls, as the MECP2 gene is located on the X chromosome. With only a single copy of this chromosome, boys are affected in devastating ways by the mutation and rarely survive early infancy. The MECP2 gene encodes methyl-CpG-binding protein-2 ( MeCP2), a transcriptional regulator of many genes including brain-derived neurotrophic factor ( BDNF ). Decreased levels of BDNF are associated with a number of neurological disorders, such as depression, schizophrenia, Alzheimer's disease and Huntington's disease. BDNF levels have also ...