Highly efficient Cas9-mediated transcriptional programming.

ABSTRACT: The RNA-guided nuclease Cas9 can be reengineered as a programmable transcription factor. However, modest levels of gene activation have limited potential applications. We describe an improved transcriptional regulator obtained through the rational design of a tripartite activator, VP64-p65-Rta (VPR), fused to nuclease-null Cas9. We demonstrate its utility in activating endogenous coding and noncoding genes, targeting several genes simultaneously and stimulating neuronal differentiation of human induced pluripotent stem cells (iPSCs).

SUBMITTER: Chavez A 

PROVIDER: S-EPMC4393883 | biostudies-literature | 2015 Apr

REPOSITORIES: biostudies-literature