RNA-guided gene activation by CRISPR-Cas9–based transcription factors

Technologies for engineering synthetic transcription factors have enabled many advances in medicine and science. In contrast to existing methods based on engineering of new DNA-binding proteins, we created a Cas9-based transactivator that is targeted to DNA sequences by guide RNA molecules. Co-expression 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.