Congratulations to Shawn Liu, Hao Wu, John Graef, and collaborators at the Whitehead Institute and Fulcrum Therapeutics for their recent Cell paper on fragile X syndrome.
Fragile X syndrome is the most frequent cause of intellectual disability in males, affecting 1 out of 3600 newborn boys. Fragile X syndrome is caused by mutations in the FMR1 gene on the X chromosome, which prevent the gene's expression. This absence of the FMR1-encoded protein during brain development has been shown to cause the overexcitability in neurons associated with the syndrome. Liu et al. demonstrate how fragile X syndrome neurons can be rescued by CRISPR-mediated DNA methylation editing of the FMR1 gene using the Maestro MEA system.
Read the paper here.
Learn more about how Maestro MEA technology can be used to study neural disease models here.