CRISPR/Cas9 technology has transformed our ability to manipulate the genome and epigenome, from efficient genomic editing to targeted localization of effectors to specific loci. Through the manipulation of DNA- and histone-modifying enzyme activities, activation or repression of gene expression, and targeting of transcriptional regulators, the role of gene-regulatory and epigenetic pathways in basic biology and disease processes can be directly queried. Here, we discuss emerging CRISPR-based methodologies, with specific consideration of neurobiological applications of human induced pluripotent stem cell (hiPSC)-based models.

中文翻译：

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CRISPR / Cas9在脑发育和神经精神疾病研究中的应用。

CRISPR / Cas9技术已经改变了我们操作基因组和表观基因组的能力，从有效的基因组编辑到效应子对特定基因座的靶向定位。通过操纵DNA和组蛋白修饰酶的活性，激活或抑制基因表达以及靶向转录调节剂，可以直接查询基因调节和表观遗传途径在基础生物学和疾病过程中的作用。在这里，我们讨论了基于CRISPR的新兴方法，特别考虑了人类诱导的多能干细胞（hiPSC）模型的神经生物学应用。