Sangamo Therapeutics, Inc.

Sangamo Biosciences, Inc.

University of Southern California

Jianbin Wang

Fyodor Urnov

Michael C Holmes

Edward J Rebar

H Steve Zhang

Gregory J Cost

Andreas Reik

Russell DeKelver

Anthony Conway

Jeffrey C Miller

Philip D Gregory

Paula M Cannon

Disclosed herein are methods and compositions for generating a single-stranded break in a target sequence, which facilitates targeted integration of one or more exogenous sequences.

Methods and compositions for targeted single-stranded cleavage and targeted integration

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

Delivery methods and compositions for nuclease-mediated genome engineering

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.

Methods and compositions for treatment of a genetic condition

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.

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.

Methods and compositions comprising single guide RNAs targeted to an endogenous mammalian &#x3b2; 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 are provided.

The present disclosure is in the field of genome engineering, particularly targeted integration of anti-HIV transgenes into the genome of a cell for the treatment and/or prevention of HIV.

Gene editing for HIV gene therapy

Disclosed herein are compositions and methods for generating chromosomal translocations and targeted deletions of specific lengths and at specific locations the genome of cell.

Methods and compostions for targeted genomic deletion

Disclosed herein are methods and compositions for modulating activity of CXCR4 genes, for example using zinc finger transcription factors (ZF-TFs) or zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding ZF-TFs or ZFNs, vectors comprising polynucleotides encoding ZF-TFs or ZFNs and cells comprising polynucleotides encoding ZF-TFs or ZFNs and/or cells comprising ZF-TF or ZFNs are also provided.

Modification of CXCR4 using engineered zinc finger proteins

Disclosed herein are methods and compositions for inhibiting viral entry into cells.

Growing community of inventors

Richmond, CA, United States of America

Jianbin Wang