AAV Split Cas9 Genome Editing and Transcriptional Regulation

摘要

The invention provides methods of altering a target nucleic acid in a cell using the AAV split Cas9 platform. The methods comprise providing the cell an enzymatically active Cas9 and optionally a transcriptional regulator fused thereto and guide RNA having different spacer sequence lengths wherein the guide RNA directs the enzymatically active Cas9 and optionally a transcriptional regulator fused thereto to either cleave a target nucleic acid or regulate expression of a target nucleic acid.