H1 CRISPR RNA COMPOSITIONS AND METHODS FOR THE EXPRESSION OF CRISPR GUIDE RNAS USING THE H1 PROMOTER

摘要

Described herein are compositions and methods for expressing CRISPR guide RNA using the H1 promoter. In particular, compositions and methods for the use of the H1 promoter to express CRISPR guide RNA (gRNA) with modified specificity of 5 'nucleotides and the use of H1 promoter sequences as bidirectional promoters to simultaneously express Cas9 nuclease and gRNA / RTI Compositions and methods are also provided for the expression and modulation of in vivo gRNA expression through the use of RNA ribozymes and regulatable aptamers.