Article
Profiling beneficial and potential adverse effects of MeCP2 overexpression in a hypomorphic Rett syndrome mouse model.
Genes, brain, and behavior - 1 Jan 2022
Vermudez Sheryl Anne D, Gogliotti Rocco G, Arthur Bright, Buch Aditi, Morales Clarissa, Moxley Yuta, Rajpal Hemangi, Conn P Jeffrey, Niswender Colleen M
Abstract excerpt
De novo loss-of-function mutations in methyl-CpG-binding protein 2 (MeCP2) lead to the neurodevelopmental disorder Rett syndrome (RTT). Despite promising results from strategies aimed at increasing MeCP2 levels, additional studies exploring how hypomorphic MeCP2 mutations impact the therapeutic window are needed. Here, we investigated the consequences of genetically introducing a wild-type MECP2 transgene in the...
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