Autism Research lectures are open to the public and are held at the Gerald D. Fischbach Auditorium at the Simons Foundation headquarters in New York City. Tea is served prior to each lecture.
In this lecture, Gail Mandel will provide a general introduction to Rett syndrome (RTT), a neurodevelopmental disease of girls that results from defects in the gene encoding of the transcription factor methyl-CpG-binding protein 2 (MeCP2). She will provide evidence that the pathology is complex, involving defects in both neurons and astrocytes in conventional RTT mouse models. She will discuss emerging ideas about the normal function of the MeCP2 protein and her recent findings that point to a role for MeCP2 in 3-D chromatin architecture. Finally, she will discuss whether Rett syndrome could be amenable to gene replacement strategies.