...Indumathy Jagadeeswaran; Jiyoung Oh; Sarah E. Sinnett Background: Rett syndrome (RTT) is a neurodevelopmental disorder caused by mutations in the transcriptional regulator methyl-CpG-binding protein 2 (MeCP2). After gene transfer in mice, exogenous MeCP2 expression must be regulated to avoid dose...

... (Rett syndrome, fragile X syndrome, and Angelman syndrome), as well as 2 less common NDDs ( SLC13A5 deficiency disorder and SLC6A1 -related disorder). We will review the available natural history of each disease and current state of preclinical studies including a discussion on the application of AAV...