Sangamo Therapeutics, Inc.

Sangamo Biosciences, Inc.

Ospedale San Raffaele S.r.l.

University of California

David Paschon

Lei Zhang

Edward J Rebar

Philip D Gregory

Jeffrey C Miller

Fyodor Urnov

Michael C Holmes

Siyuan Tan

Andreas Reik

Phillip Tam

Gregory J Cost

Luigi Naldini

Gary K Lee

Maria Chiara Bonini

Pietro Genovese

Xiangdong Meng

Russell DeKelver

Matthew C Mendel

Dmitry Y Guschin

Anthony Conway

Josee Laganiere

Elena Provasi

H Steve Zhang

Noriyuki Kasahara

Thomas Wechsler

Bryan Zeitler

Steven Froelich

Zulma Magnani

Sara Mastaglio

Dmitry M Guschin

Russell DeKeiver

Carolyn Dent

Steve H Zhang

Sumiti Jain

Russell Dekelver

Nucleases and methods of using these nucleases for alteration of a CFTR gene and generation of cells and animal models.

Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (CFTR) gene

Disclosed herein are compositions for linking DNA binding domains and cleavage domains (or cleavage half-domains) to form non-naturally occurring nucleases with alternative configurations. Also described are methods of making and using compositions comprising these linkers.

Compositions for linking DNA-binding domains and cleavage domains

Disclosed herein are polypeptides, polynucleotides encoding, 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.

DNA-binding proteins and uses thereof

Disclosed herein are methods and compositions for targeted integration of an exogenous sequence into the human PPP1R12C locus, for example, for expression of a polypeptide of interest.

Targeted integration into the PPP1R12C locus

Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins involved in clotting into the genome of a cell for treating conditions including hemophilias.

Methods and compositions for treating hemophilia

Disclosed herein are methods and compositions for inactivating TCR and/or HLA genes, using engineered nucleases comprising at least one DNA binding domain and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding nucleases, vectors comprising polynucleotides encoding nucleases and cells comprising polynucleotides encoding nucleases and/or cells comprising nucleases are also provided.

Targeted disruption of T cell and/or HLA receptors

Methods and compositions for a genetic disease are provided.

Methods and compositions for treatment of a genetic condition

Disclosed herein are methods and compositions for inactivating TCR genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided. Disclosed herein are also methods and compositions for expressing a functional exogenous TCR in the absence of endogenous TCR expression in T lymphocytes, including lymphocytes with a central memory phenotype. Polynucleotides encoding exogenous TCR, vectors comprising polynucleotides encoding exogenous TCR and cells comprising polynucleotides encoding exogenous TCR and/or cells comprising exogenous TCR are also provided.

Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases

Disclosed herein are methods and compositions for inactivating TCR genes, using engineered nucleases comprising at least one DNA binding domain and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding nucleases, vectors comprising polynucleotides encoding nucleases and cells comprising polynucleotides encoding nucleases and/or cells comprising nucleases are also provided. Disclosed herein are also methods and compositions for expressing a functional exogenous TCR in the absence of endogenous TCR expression in T lymphocytes, including lymphocytes with a central memory phenotype. Polynucleotides encoding exogenous TCR, vectors comprising polynucleotides encoding exogenous TCR and cells comprising polynucleotides encoding exogenous TCR and/or cells comprising exogenous TCR are also provided.

Targeted disruption of the T cell receptor

Disclosed herein are methods and compositions for inactivating MEW genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided.

Targeted disruption of the MHC cell receptor

Disclosed herein are methods and compositions for modulating expression of a PD1 gene.

Methods and compositions for modulating PD1

Disclosed herein are Htt repressors and methods and compositions for use of these Htt repressors.

Htt repressors and uses thereof

Disclosed herein are compositions for linking DNA binding domains and cleavage domains (or cleavage half-domains) to form non-naturally occurring nucleases. Also described are methods of making and using compositions comprising these linkers.

Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease.

Methods and compositions for genome engineering

Described herein are compositions and methods for modulation of gene expression in the liver including modulation of PCSK9, TTR, SERPINA1, KLKB1 and/or HAO1.

Growing community of inventors

Richmond, CA, United States of America

David Paschon