Siniscalco Dario, Bradstreet James Jeffrey, Sych Nataliia, Antonucci Nicola
Department of Experimental Medicine, Second University of Naples, Via S. Maria di Costantinopoli, 16-80138 Napoli, Italy ; Centre for Autism, La Forza del Silenzio, 81036 Caserta, Italy ; Cancellautismo, 50132 Florence, Italy.
Stem Cells Int. 2013;2013:262438. doi: 10.1155/2013/262438. Epub 2013 Oct 10.
Autism and autism spectrum disorders (ASDs) are complex neurodevelopmental disorders. ASDs are clinically defined by deficits in communication, social skills, and repetitive and/or restrictive interests and behaviours. With the prevalence rates for ASDs rapidly increasing, the need for effective therapies for autism is a priority for biomedical research. Currently available medications do not target the core symptoms, can have markedly adverse side-effects, and are mainly palliative for negative behaviours. The development of molecular and regenerative interventions is progressing rapidly, and medicine holds great expectations for stem cell therapies. Cells could be designed to target the observed molecular mechanisms of ASDs, that is, abnormal neurotransmitter regulation, activated microglia, mitochondrial dysfunction, blood-brain barrier disruptions, and chronic intestinal inflammation. Presently, the paracrine, secretome, and immunomodulatory effects of stem cells would appear to be the likely mechanisms of application for ASD therapeutics. This review will focus on the potential use of the various types of stem cells: embryonic, induced pluripotential, fetal, and adult stem cells as targets for ASD therapeutics.
自闭症和自闭症谱系障碍（ASD）是复杂的神经发育障碍。ASD在临床上的定义是沟通、社交技能以及重复和/或局限兴趣与行为方面存在缺陷。随着ASD的患病率迅速上升，开发有效的自闭症治疗方法成为生物医学研究的当务之急。目前可用的药物并不针对核心症状，可能会有明显的不良副作用，并且主要是缓解负面行为。分子和再生干预措施的研发进展迅速，医学界对干细胞疗法寄予厚望。可以设计细胞来针对观察到的ASD分子机制，即神经递质调节异常、小胶质细胞激活、线粒体功能障碍、血脑屏障破坏以及慢性肠道炎症。目前，干细胞的旁分泌、分泌组和免疫调节作用似乎是用于ASD治疗的可能机制。本综述将聚焦于各类干细胞（胚胎干细胞、诱导多能干细胞、胎儿干细胞和成体干细胞）作为ASD治疗靶点的潜在用途。
