RNA-Guided Human Gene Activation by CRISPR/Cas9-Based Engineered Transcription Factors
Synthetic transcription factors are central components of many applications in biotechnology, medicine, and basic research. In contrast to current methods based on engineering new DNA-binding proteins, we show that Cas9 fused to a transcriptional activation domain can be targeted by combinations of guide RNA molecules to induce the expression of endogenous human genes. In addition, Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR/Cas9-based system are described for altering gene expression and genome engineering. This straightforward and versatile approach for targeted gene activation circumvents the need for engineering new proteins and will enable widespread synthetic gene regulation.