Sangamo Therapeutics, Inc.

Sangamo Biosciences, Inc.

The University of Texas System

University of California

Massachusetts Institute of Technology

Chdi Foundation, Inc.

Edward J Rebar

Jeffrey C Miller

David Paschon

Fyodor Urnov

Philip D Gregory

Andrew Jamieson

H Steve Zhang

Lei Zhang

Qiang Liu

Stephen P Eisenberg

Andreas Reik

Michael C Holmes

Gregory J Cost

Casey Christopher Case

Siyuan Tan

Eric E Jarvis

Alan P Wolffe

Anthony Conway

Pei-Qi Liu

George N Cox

Jianbin Wang

Russell DeKelver

Dmitry Y Guschin

Trevor Collingwood

Laurence J N Cooper

Bryan Zeitler

Steven Froelich

Noriyuki Kasahara

Thomas Wechsler

Josee Laganiere

Gary K Lee

Carl O Pabo

Guofu Li

Mylavarapu Venkatramesh

Laurence Jn Cooper

Elizabeth J Wolffe

Ignacio Munoz-Sanjuan

Friedrich Fauser

Sumiti Jain

George Cox, Iii

Sean M Brennan

Laurence J Cooper

Steve Zhang H

Dmitry Guschin

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

Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of a lysosomal storage disease.

Methods and compositions for the treatment of lysosomal storage diseases

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.

Methods and compositions for regulation of transgene expression

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

Regulation of gene expression using engineered nucleases

Described herein are DNA-editing complexes, particularly DNA-editing complexes that specifically alter a single base pair in target DNA sequence as well as methods of making and using these DNA-editing complexes.

Engineered target specific base editors

Described herein are engineered nucleases comprising mutations in the cleavage domain (e.g., FokI or homologue thereof) and/or DNA binding domain (zinc finger protein, TALE, single guide RNA) such that on-target specificity is increased.

Engineered target specific nucleases

Disclosed herein are compositions comprising non-naturally occurring zinc finger domains, fusion proteins comprising these zinc finger domains, polynucleotides encoding these proteins, cells expressing these proteins and pharmaceutical compositions comprising these proteins or polynucleotides as well as methods of modifying an Htt gene using these compositions for treating or preventing Huntington's Disease.

Methods and compositions for treating Huntington's Disease

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 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

The present disclosure is in the field of modulation of Htt gene expression and HTT protein levels.

Zinc finger protein compositions for modulation of huntingtin (Htt)

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

Htt repressors and uses thereof

Method for the treatment of lysosomal storage diseases

Growing community of inventors

Richmond, CA, United States of America

Edward J Rebar