Autism spectrum disorder: prospects for treatment using gene therapy.

摘要: Autism spectrum disorder (ASD) is characterised by the concomitant occurrence of impaired social interaction; restricted, perseverative and stereotypical behaviour; abnormal communication skills. Recent epidemiological studies have reported a dramatic increase in prevalence ASD with as many 1 every 59 children being diagnosed ASD. The fact that appears to be principally genetically driven, may reversible postnatally, has raised exciting possibility using gene therapy disease-modifying treatment. Such therapies already started seriously impact on human disease particularly monogenic disorders (e.g. metachromatic leukodystrophy, SMA type 1). In regard ASD, technical advances both our capacity model animals also ability deliver genes central nervous system (CNS) led first preclinical involving replacement silencing. Furthermore, increasing awareness understanding common dysregulated pathways broadened therapy’s potential scope include various polygenic ASDs. As this review highlights, despite number outstanding challenges, excellent address cognitive dysfunction