Abstract: Disclosed is a method for assessing the transduction efficiency and/or specificity of vectors at single cell level.

Abstract: Methods of editing target nucleic acids are provided using a guide RNA and a Cas9 protein to excise exons in a target gene and where the edited gene is expressed to produce a truncated polypeptide.

Abstract: The present application discloses methods useful for the multiplex measurement of enzymatic activities. Also disclosed are polynucleotide constructs, constructs libraries and compartments related suitable for use in the methods.

Abstract: Methods of editing target nucleic acids are provided using a guide RNA and a Cas9 protein to excise exons in a target gene and where the edited gene is expressed to produce a truncated polypeptide.

Abstract: The present invention refers to a fusion protein or a protein complex comprising a DNA binding protein (DnaBP), a nucleobase modifying protein (NMP), and a Base Excision Repair associated protein (BERAP. Also, described herein are a method of replacing a cytosine with a guanine on a DNA strand in a cell and a method of treating a subject having or suspected of having a disease or disorder.

Abstract: Methods of editing target nucleic acids are provided using a guide RNA and a Cas9 protein to excise exons in a target gene and where the edited gene is expressed to produce a truncated polypeptide.

Abstract: 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.

Abstract: Split Cas9 proteins, including an active nuclease, a nickase, and a nuclease-null Cas9 protein, are provided. The Cas9 proteins were derived from nucleic acids encoding the S. pyogenes Cas9 protein. The split Cas9 proteins are provided as a first portion comprising the N-terminal lobe and a second portion comprising the C-terminal lobe. Expression of the split Cas9 proteins utilizes a split intein expression and splicing system derived from Rhodothermus marinus Methods of utilizing the split Cas9 proteins and nucleic acids encoding them for genomic engineering are presented.

Abstract: Methods of editing target nucleic acids are provided using a guide RNA and a Cas9 protein to excise exons in a target gene and where the edited gene is expressed to produce a truncated polypeptide.