Modeling Rett Syndrome Using TALEN-Edited MECP2 Mutant Cynomolgus Monkeys
Sign Up to like & getrecommendations! Published in 2017 at "Cell"
DOI: 10.1016/j.cell.2017.04.035
Abstract: Gene-editing technologies have made it feasible to create nonhuman primate models for human genetic disorders. Here, we report detailed genotypes and phenotypes of TALEN-edited MECP2 mutant cynomolgus monkeys serving as a model for a neurodevelopmental… read more here.
Keywords: talen edited; cynomolgus monkeys; rett syndrome; mutant cynomolgus ... See more keywords
Pharmacological enhancement of KCC2 gene expression exerts therapeutic effects on human Rett syndrome neurons and Mecp2 mutant mice
Sign Up to like & getrecommendations! Published in 2019 at "Science Translational Medicine"
DOI: 10.1126/scitranslmed.aau0164
Abstract: KCC2 expression–enhancing small molecules rescue cellular and behavior deficits in human RTT neurons and Mecp2 mutant mice. Restoring balance in Rett syndrome Rett syndrome (RTT) is a neurodevelopmental disorder causing cognitive, motor, and emotional impairments.… read more here.
Keywords: rtt; rett syndrome; expression; mecp2 mutant ... See more keywords