HDAC inhibitors rescue MeCP2T158Mspeckles in a high content screen
Rodrigo Lata,
Liesbeth Steegmans,
Ranie Kellens
et al.
Abstract:Rett syndrome (OMIM 312750) is a rare neurodevelopmental disorder caused byde novomutations in the Methyl-CpG Binding Protein 2 (MeCP2) gene located on the X-Chromosome, typically affecting girls. Currently, available therapy for Rett Syndrome is only symptomatic. Rett syndrome symptoms first appear between 6 to 18 months of age, characterized by microcephaly and lack of motor coordination being the most prevalent. The disease continues to progress until adulthood when it reaches a stationary phase. More than … Show more
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