Novel genetic target for fragile X syndrome could improve brain function Using an unbiased molecular screen, researchers found a new gene that could be targeted to treat fragile X syndrome. Written byAllison Whitten, PhD

https://www.drugdiscoverynews.com/novel-genetic-target-for-fragile-x-syndrome-could-improve-brain-function-17189 Fragile X syndrome (FXS) — the most commonly inherited cause of both intellectual disability and autism spectrum disorder — results from a mutation in the FMR1 gene (Fragile X messenger ribonucleoprotein 1). Though it’s just a single gene and should theoretically be a great candidate for gene therapy, preclinical and translational work targeting the FMR1 gene has so far not led to a successful treatment of the disorder.