Abstract: The application provides improved methods of genome editing. The genome editing systems described herein comprise a RNA-guided nuclease molecule and a Repair-Modulating Enzyme Molecule (RMEM).

Abstract: Compositions and methods for treatment of CEP290 related diseases are disclosed.

Abstract: Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease.

Abstract: CRISPR/CAS-related compositions and methods for treatment of Leber's Congenital Amaurosis 10 (LCA10) are disclosed.

Abstract: CRISPR/CAS-related compositions and methods for treatment of Sickle Cell Disease (SCD) are disclosed.

Abstract: Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.

Abstract: CRISPR/RNA-guided nuclease-related compositions and methods for treatment of A1AT deficiency and associated conditions are disclosed.

Abstract: The present disclosure relates to methods of assessing a sample of guide RNAs (gRNAs).

Abstract: Disclosed herein are methods for evaluation, selection, optimization, and design of Cas9 molecule/gRNA molecule complexes.

Abstract: Compositions and methods for treatment of CEP290 related diseases are disclosed.

Abstract: The methods and compositions described herein surprisingly increase CRISPR/Cas-mediated gene editing in stem cells by transiently treating the cells with a stem cell viability enhancer prior to and/or after contacting the cells with one or more CRISPR/Cas9 components. Further, this treatment also surprisingly results in increased engraftment of the stem cells into the target tissue of a subject. The present disclosure also provides one or more modified CRISPR/Cas9 components which, when used in combination with the stem cell viability enhancer, further increases the frequency of gene editing in stem cells, increases stem cell viability, and increases stem cell engraftment.

Abstract: Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.

Abstract: Genome editing systems and genetic constructs that target a herpes simplex virus (HSV) viral gene, where the systems comprise one Cas9 molecule, and a gRNA molecule, compositions and cells comprising such genome editing systems and genetic constructs as well as methods for using the genome editing systems, genetic constructs, compositions and cells for genome engineering, and for preventing, treating or reducing HSV infection.

Abstract: Provided herein are engineered immune cells, e.g. T cells, expressing a recombinant receptor, that contain a modified transforming growth factor-beta receptor type-2 (TGFBR2) locus encoding the recombinant receptor or a portion thereof. In some aspects, the cells are engineered by targeted integration of a transgene sequence encoding the recombinant receptor or a portion thereof, at a TGFBR2 genomic locus. Also provided are cell compositions containing the engineered immune cells, nucleic acids for engineering cells, and methods, kits and articles of manufacture for producing the engineered cells, such as by targeting a transgene sequence encoding a recombinant receptor or a portion thereof for integration into a region of a TGFBR2 genomic locus. In some embodiments, the engineered cells, e.g. T cells, can be used in connection with cell therapy, including in connection with cancer immunotherapy comprising adoptive transfer of the engineered cells.

Abstract: Compositions and methods for treatment of CEP290 related diseases are disclosed.

Abstract: The present disclosure is directed to the generation of NK cells (or other lymphocytes) from induced pluripotent cells that have been derived from cells, e.g., developmentally mature T cells, and uses thereof for immunotherapy.

Abstract: CRISPR/Cas-related compositions and methods for treatment of Usher Syndrome and/or Retinitis Pigmentosa are disclosed herein.

Abstract: CRISPR/RNA-guided nuclease-related compositions and methods for treatment of RHO-associated retinitis pigmentosa, e.g., autosomal-dominant retinitis pigmentosa (adRP).

Abstract: CRISPR/CAS-related compositions and methods for treatment of Leber's Congenital Amaurosis 10 (LCA10) are disclosed.

Abstract: Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.