Department of Biomedical Engineering, Duke University, Durham, North Carolina, USA.
Nat Methods. 2013 Oct;10(10):973-6. doi: 10.1038/nmeth.2600. Epub 2013 Jul 25.
Technologies for engineering synthetic transcription factors have enabled many advances in medical and scientific research. In contrast to existing methods based on engineering of DNA-binding proteins, we created a Cas9-based transactivator that is targeted to DNA sequences by guide RNA molecules. Coexpression of this transactivator and combinations of guide RNAs in human cells induced specific expression of endogenous target genes, demonstrating a simple and versatile approach for RNA-guided gene activation.
工程合成转录因子的技术已经在医学和科学研究方面取得了许多进展。与基于 DNA 结合蛋白工程的现有方法不同，我们创建了一种基于 Cas9 的转录激活子，该激活子通过向导 RNA 分子靶向 DNA 序列。在人细胞中共表达这种转录激活子和向导 RNA 的组合诱导内源性靶基因的特异性表达，证明了一种用于 RNA 引导基因激活的简单而通用的方法。
