Powell S K, Gregory J, Akbarian S, Brennand K J
Medical Scientist Training Program, Icahn School of Medicine at Mount Sinai, New York, NY 10029, United States; Department of Neuroscience, Icahn School of Medicine at Mount Sinai, New York, NY 10029, United States; Friedman Brain Institute, Icahn School of Medicine at Mount Sinai, New York, NY 10029, United States.
Instructional Technology Group, Icahn School of Medicine at Mount Sinai, New York, NY 10029, United States.
Mol Cell Neurosci. 2017 Jul;82:157-166. doi: 10.1016/j.mcn.2017.05.007. Epub 2017 May 23.
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.
CRISPR/Cas9技术已经改变了我们操控基因组和表观基因组的能力，从高效的基因组编辑到效应因子在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因子，可以直接探究基因调控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，我们将讨论基于CRISPR的新兴方法，并特别考虑基于人类诱导多能干细胞（hiPSC）模型的神经生物学应用。
