FMR1 Mutant Marmosets Show Fragile X Syndrome Phenotypes

Fragile X syndrome (FXS) is the leading monogenic cause of autism spectrum disorder and intellectual disability, caused by silencing of the FMR1 gene. To determine the effect of FMRP loss on the mRNA profile in a marmoset model of FXS, we performed RNA-sequencing of brain samples from neonatal wild-type and FMR1 mutant marmosets. Overall design: We generated FMR1 mutant marmosets using CRISPR/Cas9 system. We then performed gene expression profiling analysis using data obtained from RNA-seq of forebrain samples, including the cerebral cortex, striatum, and hippocampus, from wild-type and FMR1 mutant marmosets at postnatal day 5.