Genetic studies of epigenetic modifiers such as DNA methyltransferases and histone acetyltransferases have revealed a critical role for epigenetic regulation during brain development and function. Alteration of epigenetic modifications have been documented in a variety of brain disorders, including neurodevelopmental, psychiatric, and neurodegenerative diseases. Development of epigenome editing tools enables a functional dissection of the link between altered epigenetic changes and disease outcomes. Here, we review the development of epigenome editing tools, summarize proof of concept applications focusing on brain disease-associated genes, and discuss the promising application and challenges of epigenome editing to tackle brain disorders.

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编辑表观基因组来应对大脑疾病

DNA甲基转移酶和组蛋白乙酰转移酶等表观遗传修饰剂的遗传学研究揭示了表观遗传调控在大脑发育和功能过程中的关键作用。表观遗传修饰的改变已被记录在多种脑部疾病中，包括神经发育疾病、精神疾病和神经退行性疾病。表观基因组编辑工具的开发可以对表观遗传变化与疾病结果之间的联系进行功能性剖析。在这里，我们回顾了表观基因组编辑工具的发展，总结了针对脑疾病相关基因的概念验证应用，并讨论了表观基因组编辑在解决脑疾病方面的前景应用和挑战。