Modeling Autism Spectrum Disorders Using Human Neurons

摘要

The cellular and molecular mechanisms of neurodevelopmental conditions such as autism spectrum disorders (ASDs) have been studied intensively for decades. The unavailability of live patient neurons for research, however, has represented a major obstacle in the elucidation of the disease etiologies. The generation of human neurons from induced pluripotent stem cells (iPSCs) derived from patients with ASDs offers a novel and complementary opportunity for basic research and the development of therapeutic compounds aiming to revert or ameliorate the condition. The findings of relevant phenotypes in Rett syndrome iPSC-derived neurons suggest that iPSC technology offers a novel and unique opportunity for understanding and developing therapeutics for other ASDs. Neurons-in-a-dish from syndromic forms of ASD open new avenues for the stratification of different subtypes of idiopathic autism. In this chapter, I will discuss the conceptual and practical issues related to modeling ASD using human neurons.