FMR1 and fragile X syndrome: Whilethere is still much to learn about the role of FMRP in individualsand model systems, there is great motivation from researchers andfamilies to work together for the benefit of affected individuals.While gene therapy for Fragile X syndrome is still in its infancy,recent successes in other single gene conditions (such as spinal muscularatrophy) are setting the ground rules for how such therapies may beemployed and what hurdles they need to overcome.