Institute of Science and Technology Austria, Klosterneuburg, Austria.
Institute of Science and Technology Austria, Klosterneuburg, Austria.
Curr Opin Genet Dev. 2020 Dec;65:126-137. doi: 10.1016/j.gde.2020.06.004. Epub 2020 Jul 10.
The possibility to generate construct valid animal models enabled the development and testing of therapeutic strategies targeting the core features of autism spectrum disorders (ASDs). At the same time, these studies highlighted the necessity of identifying sensitive developmental time windows for successful therapeutic interventions. Animal and human studies also uncovered the possibility to stratify the variety of ASDs in molecularly distinct subgroups, potentially facilitating effective treatment design. Here, we focus on the molecular pathways emerging as commonly affected by mutations in diverse ASD-risk genes, on their role during critical windows of brain development and the potential treatments targeting these biological processes.
构建具有充分结构效度的动物模型的可能性,使得靶向自闭症谱系障碍(ASD)核心特征的治疗策略的开发和测试成为可能。与此同时,这些研究强调了确定成功治疗干预的敏感发育时间窗的必要性。动物和人类研究也揭示了在分子上明显不同的亚组中对 ASD 进行分类的可能性,这可能有助于有效的治疗设计。在这里,我们重点关注在大脑发育的关键窗口期,受多种 ASD 风险基因突变共同影响的分子途径,以及针对这些生物过程的潜在治疗方法。