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自闭症谱系障碍的新疗法:从突触功能障碍到实验治疗学。

Novel treatments in autism spectrum disorders: from synaptic dysfunction to experimental therapeutics.

机构信息

Division of Child Neuropsychiatry, University Hospital of Siena, Italy.

出版信息

Behav Brain Res. 2013 Aug 15;251:125-32. doi: 10.1016/j.bbr.2012.11.024. Epub 2012 Nov 29.

Abstract

Recent discoveries and advances in genetics and neuroscience have provided deeper understanding of the complex neurobiology of ASD. The development of novel treatments is strictly dependent on these findings in order to design new strategies in the pharmacotherapy of ASD. At this time, therapeutics are limited to treating associated core, symptoms. Studies of single gene disorders, such as Phelan-McDermid syndrome, Fragile X and Tuberous Sclerosis, might be of significant help since the neurobiology of these disorders is clearer and clinical trials are already underway for these conditions. The pathogenesis paradigm shift of ASD towards synaptic abnormalities has led to current research of the pathways to disease, which involves multiple dynamic systems. Interest in oxytocin is growing as it has been recognized to be implicated in social development and affiliative behaviours. In the future, progress is expected in possible new options for therapeutics in ASD. Children and adolescents with ASD and their families can provide vital information about their experiences with new treatments, which should be a priority for future research.

摘要

最近在遗传学和神经科学方面的发现和进展,为理解 ASD 的复杂神经生物学提供了更深入的认识。新型治疗方法的开发严格依赖于这些发现,以便在 ASD 的药物治疗中设计新策略。此时,治疗方法仅限于治疗相关的核心症状。对单一基因疾病的研究,如 Phelan-McDermid 综合征、脆性 X 综合征和结节性硬化症,可能会有很大帮助,因为这些疾病的神经生物学更为明确,并且已经针对这些疾病开展了临床试验。ASD 的发病机制从突触异常到疾病的病理途径发生了转变,这涉及到多个动态系统。由于人们已经认识到催产素与社会发展和亲和行为有关,因此对其的兴趣日益增加。未来,有望在 ASD 的治疗方法上取得新的进展。患有 ASD 的儿童和青少年及其家庭可以提供有关新治疗方法的宝贵经验,这应该是未来研究的重点。

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