Abstract: Disclosed herein are methods and compositions for targeted integration of a exogenous sequence into a predetermined target site in a genome for use, for example, in protein expression and gene inactivation.
Type:
Application
Filed:
March 6, 2013
Publication date:
September 19, 2013
Applicant:
SANGAMO BIOSCIENCES, INC.
Inventors:
Philip D. Gregory, Michael C. Holmes, Edward J. Rebar, Fyodor Urnov
Abstract: Disclosed herein are methods and compositions for targeted cleavage of a genomic sequence, targeted alteration of a genomic sequence, and targeted recombination between a genomic region and an exogenous polynucleotide homologous to the genomic region. The compositions include fusion proteins comprising a cleavage domain (or cleavage half-domain) and an engineered zinc finger domain and polynucleotides encoding same. Methods for targeted cleavage include introduction of such fusion proteins, or polynucleotides encoding same, into a cell. Methods for targeted recombination additionally include introduction of an exogenous polynucleotide homologous to a genomic region into cells comprising the disclosed fusion proteins.
Type:
Grant
Filed:
July 16, 2010
Date of Patent:
September 3, 2013
Assignee:
Sangamo BioSciences, Inc.
Inventors:
Fyodor Urnov, Michael C. Holmes, Jeffrey C. Miller, Carl O. Pabo
Abstract: Disclosed herein are methods and compositions for inactivating CCR-5 genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs, such as adenovirus (Ad) vectors, and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided.
Type:
Grant
Filed:
May 5, 2011
Date of Patent:
September 3, 2013
Assignee:
Sangamo BioSciences, Inc.
Inventors:
Dale Ando, Michael C. Holmes, Yann Jouvenot, Gary Ka Leong Lee
Abstract: Disclosed herein are methods and compositions for genome editing of the malarial parasite Plasmodium, and for the use of the edited Plasmodium in the development of vaccines and therapeutics.
Type:
Application
Filed:
January 23, 2013
Publication date:
August 22, 2013
Applicants:
The Trustees of Columbia University in the City of New York, Sangamo BioSciences, Inc.
Inventors:
Sangamo BioSciences, Inc., The Trustees of Columbia University in the City of New York
Abstract: Disclosed herein are enhanced polypeptides, polynucleotides encoding these polypeptides, cells and organisms comprising novel DNA-binding domains, including TALE DNA-binding domains. Also disclosed are methods of using these novel DNA-binding domains for modulation of gene expression and/or genomic editing of endogenous cellular sequences.
Abstract: Nucleases and methods of using these nucleases for expressing a transgene from a safe harbor locus in a secretory tissue, and clones and animals derived therefrom.
Abstract: Nucleases and methods of using these nucleases for expressing a transgene from a safe harbor locus in a secretory tissue, and clones and animals derived therefrom.
Abstract: Disclosed herein are methods and compositions for rapidly identifying active nucleases and cells having nuclease-mediated genomic modifications.
Abstract: Methods and compositions for regulating HIV infection and/or replication in which an anti-HIV transgene is integrated into the genome of a cell using a nuclease.
Abstract: A variety of zinc finger proteins (ZFPs) and methods utilizing such proteins are provided for use in treating neuropathic pain. ZFPs that bind to a target site in genes that are aberrantly expressed in subjects having neuropathic pain are described. In addition, ZFPs that bind to a target site in genes expressed at normal levels in subjects experiencing neuropathic pain, modulation of whose expression results in decreased pain perception, are also provided. For example, genes that are over-expressed in the dorsal root ganglia (DRG) of pain patients (e.g., VR1, TRKA and/or Nav1.8) can be repressed, whereas genes that are under-expressed in the same populations can be activated.
Abstract: The present disclosure relates to engineered zinc finger proteins that target 5-enolpyruvyl shikimate-3-phosphate synthase (EPSPS) genes in plants and methods of using such zinc finger proteins in modulating gene expression, gene inactivation, and targeted gene modification. In particular, the disclosure pertains to zinc finger nucleases for targeted cleavage and alteration of EPSPS genes.
Abstract: Nucleases and methods of using these nucleases for modification of an HPRT locus and for increasing the frequency of gene modification at a targeted locus and clones and for generating animals.
Type:
Application
Filed:
October 25, 2012
Publication date:
May 30, 2013
Applicants:
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA, SANGAMO BIOSCIENCES, INC.
Inventors:
SANGAMO BIOSCIENCES, INC., THE REGENTS OF THE UNIVERSITY OF CALIF
Abstract: The specificity of binding of a zinc finger to a triplet or quadruplet nucleotide target subsite depends upon the location of the zinc finger in a multifinger protein and, hence, upon the location of its target subsite within a larger target sequence. The present disclosure provides zinc finger amino acid sequences for recognition of triplet target subsites having the nucleotide G in the 5?-most position of the subsite, that have been optimized with respect to the location of the subsite within the target site. Accordingly, the disclosure provides finger position-specific amino acid sequences for the recognition of GNN target subsites. This allows the construction of multi-finger zinc finger proteins with improved affinity and specificity for their target sequences, as well as enhanced biological activity.
Abstract: Nucleases and methods of using these nucleases for modification of an HPRT locus and for increasing the frequency of gene modification at a targeted locus and clones and for generating animals.
Type:
Application
Filed:
October 25, 2012
Publication date:
May 16, 2013
Applicants:
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA, SANGAMO BIOSCIENCES, INC.
Inventors:
Sangamo BioSciences, Inc., The Regents of the University of California
Abstract: Disclosed herein are methods and compositions for targeted deletion of double-stranded DNA. The compositions include fusion proteins comprising a cleavage domain (or cleavage half-domain) and an engineered zinc finger domain, and polynucleotides encoding same. Methods for targeted deletion include introduction of such fusion proteins, or polynucleotides encoding same, into a cell such that two targeted cleavage events occur. Subsequent cellular repair mechanisms result in deletion of sequences between the two cleavage sites.
Abstract: The specificity of binding of a zinc finger to a triplet or quadruplet nucleotide target subsite depends upon the location of the zinc finger in a multifinger protein and, hence, upon the location of its target subsite within a larger target sequence. The present disclosure provides zinc finger amino acid sequences for recognition of triplet target subsites having the nucleotide G in the 5?-most position of the subsite, that have been optimized with respect to the location of the subsite within the target site. Accordingly, the disclosure provides finger position-specific amino acid sequences for the recognition of GNN target subsites. This allows the construction of multi-finger zinc finger proteins with improved affinity and specificity for their target sequences, as well as enhanced biological activity.
Type:
Grant
Filed:
August 15, 2007
Date of Patent:
February 26, 2013
Assignee:
Sangamo BioSciences, Inc.
Inventors:
Qiang Liu, Edward Rebar, Andrew C. Jamieson
Abstract: Disclosed herein are methods and compositions for targeted cleavage of a genomic sequence, targeted alteration of a genomic sequence, and targeted recombination between a genomic region and an exogenous polynucleotide homologous to the genomic region. The compositions include fusion proteins comprising a cleavage domain (or cleavage half-domain) and an engineered zinc finger domain, as well as polynucleotides encoding same. Fusion proteins comprising cleavage half-domains are used in pairs to reconstitute a functional cleavage domain. In these fusion proteins, the zinc finger domain can be N-terminal to the cleavage half-domain, or the cleavage half-domain can be N-terminal to the zinc finger domain.