公开(公告)号：WO2013044008A2

公开(公告)日：2013-03-28

申请号：PCT/US2012/056539

申请日：2012-09-21

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： REBAR, Edward J.

IPC: C12N15/85

CPC classification number: C12N15/62 ,  C07K14/00 ,  C07K2319/81 ,  C12N9/22 ,  C12N9/96 ,  C12N15/85 ,  C12N2799/025

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 translation: 使用这些核酸酶从分泌组织中的安全港位置表达转基因的核酸酶和方法，以及从其衍生的克隆和动物。

公开(公告)号：WO2014036219A2

公开(公告)日：2014-03-06

申请号：PCT/US2013/057214

申请日：2013-08-29

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： COST, Gregory J. ,  GREGORY, Philip D. ,  GUSCHIN, Dmitry ,  HOLMES, Michael C. ,  MILLER, Jeffrey C. ,  PASCHON, David ,  REBAR, Edward J. ,  REIK, Andreas ,  URNOV, Fyodor ,  ZHANG, Lei

IPC: C12N15/52 ,  C12N9/22

CPC classification number: C12N15/85 ,  C07K14/4702 ,  C07K14/805 ,  C07K2319/81 ,  C12N9/22 ,  C12N15/52

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

Abstract translation: 提供了遗传疾病的方法和组合物。

公开(公告)号：WO2013063315A2

公开(公告)日：2013-05-02

申请号：PCT/US2012/061986

申请日：2012-10-25

Applicant: SANGAMO BIOSCIENCES, INC. ,  THE REGENTS OF THE UNIVERSITY OF CALIFORNIA

Inventor： COST, Gregory J. ,  HOLMES, Michael C. ,  KASAHARA, Noriyuki ,  LAGANIERE, Josee ,  MILLER, Jeffrey C. ,  PASCHON, David ,  REBAR, Edward J. ,  URNOV, Fyodor ,  ZHANG, Lei

IPC: C12N15/85

CPC classification number: C12N15/85 ,  C07K2319/81 ,  C12N9/1077 ,  C12N9/22 ,  C12N9/96 ,  C12N15/907 ,  C12Y204/02008 ,  C12Y301/21004

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.

Abstract translation: 核酸酶和使用这些核酸酶修饰HPRT基因座并增加靶基因座和克隆的基因修饰频率以及产生动物的方法。

公开(公告)号：WO2017053753A1

公开(公告)日：2017-03-30

申请号：PCT/US2016/053374

申请日：2016-09-23

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： FROELICH, Steven ,  MILLER, Jeffrey C. ,  PASCHON, David ,  REBAR, Edward J. ,  ZEITLER, Bryan ,  ZHANG, H. Steve

IPC: A61K38/16 ,  C07K14/47 ,  C12Q1/68

CPC classification number: C07K14/47 ,  C07K14/4702 ,  C07K2319/81

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

Abstract translation: 本文公开了使用这些Htt阻遏物的Htt阻遏物和方法和组合物。

公开(公告)号：WO2015089046A1

公开(公告)日：2015-06-18

申请号：PCT/US2014/069307

申请日：2014-12-09

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： MILLER, Jeffrey C. ,  PASCHON, David ,  REBAR, Edward J.

IPC: C07K14/00

CPC classification number: C07K14/47 ,  A61K48/00 ,  C07K14/4702 ,  C07K2319/81 ,  C12N9/22 ,  C12N9/644 ,  C12N15/86 ,  C12N2750/14133 ,  C12N2750/14142 ,  C12N2750/14143

Abstract: 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. In one aspect, described herein is a non-naturally occurring zinc-finger protein (ZFP) that binds to a target site in a region of interest (e.g., an albumin gene) in a genome, wherein the ZFP comprises one or more engineered zinc-finger binding domains. In certain embodiments, the zinc finger domain recognizes a target site in an albumin gene. In some embodiments, the zinc finger protein comprises five or six zinc finger domains designated and ordered F1 to F5 or F1 to F6, as shown in a single row of Table 1.

Abstract translation: 本文公开了用于将编码参与凝血的蛋白质的转基因序列插入细胞的基因组中用于治疗包括血友病的病症的方法和组合物。 在一个方面，本文描述了与基因组中目标区域（例如，白蛋白基因）结合的靶位点的非天然存在的锌指蛋白（ZFP），其中ZFP包含一种或多种工程化锌 指针结合域。 在某些实施方案中，锌指结构域识别白蛋白基因中的靶位点。 在一些实施方案中，锌指蛋白包含五个或六个锌指结构域，其指定并排列F1至F5或F1至F6，如表1的单行所示。

公开(公告)号：WO2012012667A3

公开(公告)日：2012-01-26

申请号：PCT/US2011/044902

申请日：2011-07-21

Applicant: SANGAMO BIOSCIENCES, INC. ,  BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM ,  COLLINGWOOD, Trevor ,  COOPER, Laurence J.,N. ,  GREGORY, Philip D. ,  HOLMES, Michael C. ,  MILLER, Jeffrey C. ,  REBAR, Edward J. ,  REIK, Andreas ,  URNOV, Fyodor

Inventor： COLLINGWOOD, Trevor ,  COOPER, Laurence J.,N. ,  GREGORY, Philip D. ,  HOLMES, Michael C. ,  MILLER, Jeffrey C. ,  REBAR, Edward J. ,  REIK, Andreas ,  URNOV, Fyodor

IPC: C07K14/00

Abstract: Disclosed herein are methods and compositions for modulating the expression of a HLA locus or for selectively deleting or manipulating a HLA locus or HLA regulator.

公开(公告)号：WO2007014275A2

公开(公告)日：2007-02-01

申请号：PCT/US2006/029027

申请日：2006-07-26

Applicant: SANGAMO BIOSCIENCES, INC. ,  HOLMES, Michael C. ,  URNOV, Fyodor ,  GREGORY, Philip D. ,  REBAR, Edward J. ,  BRENNAN, Sean M.

Inventor： HOLMES, Michael C. ,  URNOV, Fyodor ,  GREGORY, Philip D. ,  REBAR, Edward J. ,  BRENNAN, Sean M.

IPC: C12N15/09

CPC classification number: C12N15/907 ,  A61K48/0008 ,  A61K48/005 ,  A61K48/0058 ,  C07K2319/00 ,  C07K2319/81 ,  C12N9/22 ,  C12N15/62 ,  C12N15/90

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.

Abstract translation: 本文公开了用于将外源序列靶向整合到基因组中的预定靶位点以用于例如蛋白质表达和基因失活的方法和组合物。

公开(公告)号：WO2017136049A1

公开(公告)日：2017-08-10

申请号：PCT/US2016/066999

申请日：2016-12-15

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： MILLER, Jeffrey C. ,  PASCHON, David ,  REBAR, Edward J.

IPC: A61K38/00 ,  A61K38/46 ,  C07K19/00 ,  C12N9/16 ,  C12N9/22

CPC classification number: C12N9/22 ,  C12N15/52 ,  C12N15/62 ,  C12N15/907 ,  C12Y301/00

Abstract: 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.

Abstract translation: 本文公开了用于连接DNA结合结构域和切割结构域（或切割半结构域）以形成具有可选构型的非天然存在的核酸酶的组合物。 还描述了制造和使用包含这些接头的组合物的方法。

公开(公告)号：WO2015164748A1

公开(公告)日：2015-10-29

申请号：PCT/US2015/027524

申请日：2015-04-24

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： MILLER, Jeffrey C. ,  REBAR, Edward J.

IPC: C07K14/195

CPC classification number: C07K14/195 ,  C07K2319/00 ,  C07K2319/80 ,  C12N9/22 ,  C12N15/902

Abstract: Disclosed herein are methods and compositions for design and use of engineered TALEs. The present invention describes TALE compositions and methods for use in gene therapy and genome engineering. Specifically, the methods and compositions described relate to TALE proteins with RVDs that show enhanced 5 activity and/or specificity as compared to previously-described TALEs using exclusively canonical RVDs, for example, by substantially reducing off-target genome cleavage. By providing novel RVDs and novel strategies for design of engineered TALEs, the present disclosure establishes options for developing novel TALEs with broader application across medicine and biotechnology.

Abstract translation: 本文公开了用于设计和使用工程TALE的方法和组合物。 本发明描述了用于基因治疗和基因组工程的TALE组合物和方法。 具体地说，所描述的方法和组合物涉及具有RVDs的TALE蛋白，其与以前描述的仅使用典型RVDs的TALE相比显示增强的5活性和/或特异性，例如通过基本上减少脱靶基因组切割。 通过提供新颖的RVD和设计TALE的新策略，本公开确定了开发具有更广泛应用于医学和生物技术的新型TALE的选择。

公开(公告)号：WO2015089077A2

公开(公告)日：2015-06-18

申请号：PCT/US2014/069352

申请日：2014-12-09

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： MILLER, Jeffrey C. ,  PASCHON, David ,  REBAR, Edward J. ,  WECHSLER, Thomas ,  ZHANG, Lei

IPC: C12N15/86 ,  C12N9/22

CPC classification number: C07K14/47 ,  A61K48/00 ,  C07K14/4702 ,  C07K2319/81 ,  C12N9/22 ,  C12N9/644 ,  C12N15/86 ,  C12N2750/14133 ,  C12N2750/14142 ,  C12N2750/14143

Abstract: 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.

Abstract translation: 本文公开了用于插入编码蛋白质的转基因序列的方法和组合物，所述蛋白质在疾病或病症例如溶酶体贮积病中异常表达。