Abstract: Methods and compositions for a genetic disease are provided.

Abstract: Methods and compositions for a genetic disease are provided.

Abstract: The present application relates to compositions and methods for genome editing in cells by homology-independent mechanisms, in particular for genome editing in cells that lack the machinery necessary for repair by homology-dependent mechanisms.

Abstract: Disclosed herein are methods and compositions for enhancing insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease or a hemophilia by administering one or more topoisomerases inhibitors, one or more stabilizers of R loop formation or inhibitors of R-loop repair and/or one or more up-regulators of the TC-NER pathway to the target cell.

Abstract: Nucleases and methods of using these nucleases for genetic alteration of red blood cells (RBCs), for example for providing for a protein lacking in a monogenic disorder or a biologic for the treatment of exposure to a toxin using genetically altered RBCs.

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic stem cell.

Abstract: Methods and compositions comprising a CRISPR/Cas nuclease that cleaves a T cell receptor (TCR) gene and/or human leukocyte (HLA) gene, the nuclease comprising a cleavage domain and a single guide RNA that binds to a target site in the TCR and/or HLA gene.

Abstract: Methods and compositions comprising a CRISPR/Cas nuclease that cleaves a BCL11A gene for the modification of the BCL11A gene and for modification of globin expression.

Abstract: Methods and compositions for increasing transgene expression and/or activity, including for increasing nuclease-mediated genomic modifications.

Abstract: Disclosed herein are methods and compositions for homology-independent targeted insertion of donor molecules into the genome of a cell.

Abstract: Methods and compositions for a genetic disease are provided.

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic stem cell.

Abstract: Methods and compositions comprising single guide RNAs targeted to endogenous gene for genetic alteration of cells are provided.

Abstract: Disclosed herein are donor molecules comprising single-stranded complementary regions flanking one or more sequences of interest. The donor molecules and/or compositions comprising these molecules can be used in methods for targeted integration of an exogenous sequence into a specified region of interest in the genome of a cell.

Abstract: Disclosed herein are nucleases and methods of using these nucleases for alteration of a globin gene and generation of cells.

Abstract: Methods and compositions for comprising single guide RNAs targeted to an endogenous gene for genetic alteration of cells are provided.

Abstract: Methods and compositions comprising a fusion protein comprising a Cas protein and a functional domain in association with a single guide RNA targeted to an endogenous gene for genetic alteration of cells are provided.

Abstract: Disclosed herein are methods and compositions for inactivating a FUT8 gene, using fusion proteins comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding said fusion proteins are also provided, as are cells comprising said polynucleotides and fusion proteins.

Abstract: Methods and compositions comprising single guide RNAs targeted to an endogenous mammalian ? globin gene (HBB), gamma globin gene (HBG1), B-cell lymphoma/leukemia 11A (BCL11A) gene, Kruppel-like factor 1 (KLF1) gene, CCR5 gene, CXCR4 gene, PPP1R12C (AAVS1) gene, hypoxanthine phosphoribosyltransferase (HPRT) gene, albumin gene, Factor VIII gene, Factor IX gene, Leucine-rich repeat kinase 2 (LRRK2) gene, Hungtingin (Htt) gene, rhodopsin (RHO) gene, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, surfactant protein B gene (SFTPB), T-cell receptor alpha (TRAC) gene, T-cell receptor beta (TRBC) gene, programmed cell death 1 (PD1) gene, Cytotoxic T-Lymphocyte Antigen 4 (CTLA-4) gene, human leukocyte antigen (HLA) A gene, a LMP7 gene, a Transporter associated with Antigen Processing (TAP) gene, tapasin gene (TAPBP), a class II major histocompatibility complex transactivator (CIITA) gene, a dystrophin gene (DMD), a glucocorticoid receptor gene (GR), IL2RG gene or RFX5 for genetic alteration of cells a

Abstract: The present disclosure is in the field of genome engineering, particularly targeted integration of a functional SCID-related genes (e.g., IL2RG, RAG1 and/or RAG2 gene) into the genome of a cell for provision of proteins lacking or deficient in SCID.