Abstract: The present disclosure relates to compositions, systems, vectors, and methods for the treatment, prevention, and/or reduction of viral infection and viral infection-related diseases. In particular, the methods disclosed herein involve gene editing approaches using a genome editing system targeting a viral genome, where the expression of at least one component of the gene editing system is regulated by a promoter derived from the targeted viral family, genus, and/or species.

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: Provided are binding molecules, such as TCRs or antigen binding fragments thereof and antibodies and antigen-binding fragments thereof, such as those that recognize or bind human papilloma virus (HPV) 16, including HPV 16 E6 and HPV 16 E7. Also provided are engineered cells containing such binding molecules, compositions containing the binding molecules or engineered cells, and methods of treatment, such as administration of the binding molecules, engineered cells, or compositions.

Abstract: The present disclosure relates to Cas9 nuclease variants and methods of producing and using such variants.

Abstract: Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci in cells and increasing expression of fetal hemoglobin.

Abstract: Engineered nucleic acids encoding genome editing system components are provided, as are engineered RNA-guided nucleases that include inserts encoded in part by cellular genomic or other sequences recognized by guide RNAs.

Abstract: Disclosed herein are genome editing systems and compositions that target a cystic fibrosis transmembrane conductance regulator (CFTR) gene and a sodium channel epithelial 1 alpha (SCNN1A) gene, comprising a Cas9 molecule, and a gRNA molecule comprising a targeting domain that is complementary with a target sequence of a CFTR gene or a SCNN1A gene, and cells comprising such genome editing systems and compositions. Also provided are methods for using the genome editing systems, compositions, and cells for genome engineering (e.g., altering a CFTR gene and/or a SCNN1A gene), and for preventing or treating Cystic Fibrosis (CF) and CF-like disease.

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

Abstract: CRISPR/CAS-related systems, compositions and methods for editing RS1, RL2, and/or LAT genes in human cells are described, as are cells and compositions including cells edited according to the same.

Abstract: Methods and compositions useful in targeting a payload to or editing a target nucleic acid are disclosed herein.

Abstract: Provided herein are methods for engineering immune cells, cell compositions containing engineered immune cells, kits and articles of manufacture for targeting nucleic acid sequence encoding a portion of a recombinant receptor, e.g., a recombinant T cell receptor (TCR), to a particular genomic locus and/or for modulating expression of the gene at the genomic locus, and applications thereof in connection with cancer immunotherapy, such as adoptive transfer of engineered T cells. In some aspects, the nucleic acid sequence integrates in-frame into the locus of a receptor encoding gene, and in some aspects, results in expression of the whole recombinant receptor.

Abstract: CRISPR/CAS-related systems, compositions and methods for editing HSV-1 genes in human cells are described, as are cells and compositions including cells edited according to the same. Methods for treating HSV-related keratitis using the said CRISPR/CAS-related systems, compositions and methods are also described.

Abstract: Provided herein are methods and compositions for treating an eye disorder, for example CORD6. Aspects of the disclosure relate to knocking out an autosomal dominant mutant GUCY2D gene.

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: The present disclosure relates to CRISPR/Cpf1-related methods and components for editing a target nucleic acid sequence and/or modulating expression of a target nucleic acid sequence, as well as methods and compositions for evaluating such editing and/or modulation of expression.

Abstract: Genome editing systems, guide RNAs, DNA donor templates, and CRISPR-mediated methods are provided for altering a ?-globin gene to alter a genotype, e.g., by correcting or partially correcting, a genotype associated with thalassemia or sickle cell disease.

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

Abstract: The present disclosure relates to the manipulation of nucleic acids, and more particularly to systems and methods for nucleic acid mutagenesis.

Abstract: Genome editing systems, guide RNAs, dead guide RNAs, and CRISPR-mediated methods are provided for altering portions of a target nucleic acid.