公开(公告)号：WO2014186585A2

公开(公告)日：2014-11-20

申请号：PCT/US2014/038216

申请日：2014-05-15

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： CONWAY, Anthony ,  COST, Gregory J. ,  DEKELVER, Russell ,  REBAR, Edward J. ,  REIK, Andreas ,  URNOV, Fyodor ,  WANG, Jianbin ,  ZHANG, H. Steve

IPC: C12N15/85

CPC classification number: C12N15/907 ,  C07K2/00 ,  C07K2319/81 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90 ,  C12N15/902 ,  C12N2320/30 ,  C12Y301/21004

Abstract: Methods and compositions for genetic alteration of cells are provided.

Abstract translation: 提供了细胞遗传改变的方法和组合物。

公开(公告)号：WO2015054253A1

公开(公告)日：2015-04-16

申请号：PCT/US2014/059497

申请日：2014-10-07

Applicant: SANGAMO BIOSCIENCES, INC. ,  UNIVERSITATSKLINIKUM TUBINGEN

Inventor： COST, Gregory J. ,  KORMANN, Michael ,  MAYS, Lauren ,  ZHANG, Lei

IPC: C12N15/00 ,  C12N15/86 ,  C12N15/87 ,  C07H21/04

CPC classification number: C12N9/22 ,  C07K14/4712 ,  C07K14/785 ,  C12N15/111 ,  C12N15/115 ,  C12N2310/16 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/3519

Abstract: The disclosure is in the field of genome engineering, particularly targeted modification of the genome using a nucleotide-modified mRN (nec-mRNA). Specifically, the methods and compositions relate to introducing nuclease-encoding nucleotide (chemically)-modified messenger RNA (nec-mRNA) into cells for genomic editing of a target cell, such as a nuclease that targets a gene encoding surfactant B protein (SB-P).

Abstract translation: 本公开在基因组工程领域，特别是使用核苷酸修饰的mRN（nec-mRNA）的基因组的靶向修饰。 具体地，所述方法和组合物涉及将核酸酶编码核苷酸（化学上）修饰的信使RNA（nec-mRNA）引入用于靶细胞的基因组编辑的细胞，例如靶向编码表面活性剂B蛋白（SB- P）。

公开(公告)号：WO2014011901A2

公开(公告)日：2014-01-16

申请号：PCT/US2013/050107

申请日：2013-07-11

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： COST, Gregory J.

IPC: A61K35/18

CPC classification number: C12N15/907 ,  A61K35/18 ,  A61K38/465 ,  C12Y301/01008

Abstract: Nucleases and methods of using these nucleases for genetic alteration of red blood cells (RBCs), for example for providing for a protein lacking in a monogenic disorder or a biologic for the treatment of exposure to a toxin using genetically altered RBCs.

Abstract translation: 使用这些核酸酶进行红细胞（RBC）基因改变的核酸酶和方法，例如用于提供缺乏单基因病症的蛋白质或用于使用毒素暴露治疗的生物制剂 遗传改变的红细胞。

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：WO2016044416A1

公开(公告)日：2016-03-24

申请号：PCT/US2015/050411

申请日：2015-09-16

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： COST, Gregory J. ,  MILLER, Jeffrey C. ,  ZHANG, Lei

IPC: C12N5/071

CPC classification number: C12N9/22 ,  A61K35/28 ,  A61K48/005 ,  C07K14/805 ,  C12N5/0645 ,  C12N15/11 ,  C12N15/111 ,  C12N15/907 ,  C12N2310/20 ,  C12N2510/00 ,  C12N2800/80

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic stem cell. Disclosed herein are methods and compositions for altering the expression or for correcting one or more genes encoding proteins involved in a genetic disease (e.g., producing proteins lacking, deficient or aberrant in the disease and/or proteins that regulate these proteins) such as sickle cell disease. The present invention describes compositions and methods for use in gene therapy and genome engineering.

Abstract translation: 本公开在基因组工程领域，特别是造血干细胞基因组的靶向修饰。 本文公开了用于改变表达或用于校正编码参与遗传疾病的蛋白质的一种或多种基因的方法和组合物（例如，产生调节这些蛋白质的疾病和/或蛋白质中缺乏，缺陷或异常的蛋白质），例如镰状细胞 疾病。 本发明描述了用于基因治疗和基因组工程的组合物和方法。

公开(公告)号：WO2016014794A1

公开(公告)日：2016-01-28

申请号：PCT/US2015/041729

申请日：2015-07-23

Applicant: SANGAMO BIOSCIENCES, INC.

Inventor： CONWAY, Anthony ,  COST, Gregory J. ,  GREGORY, Philip D.

IPC: C12N15/90

CPC classification number: C12N5/0647 ,  A61K35/28 ,  C12N9/22 ,  C12N2500/38 ,  C12N2501/065 ,  C12N2501/2306 ,  C12N2501/999 ,  C12N2510/00

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic stem cell. The present invention describes compositions and methods for use in gene therapy and genome engineering, particularly of hematopoietic cells, including HSCs. The present inventors have determined that certain culture conditions can impact the efficiency and the nature of gene modification after a double strand break (independent of the nuclease used), particularly in LT-HSCs.

Abstract translation: 本公开在基因组工程领域，特别是造血干细胞基因组的靶向修饰。 本发明描述了用于基因治疗和基因组工程，特别是造血细胞（包括HSC）的组合物和方法。 本发明人已经确定某些培养条件可以影响双链断裂后的基因修饰的效率和性质（与使用的核酸酶无关），特别是在LT-HSC中。

公开(公告)号：WO2009009086A3

公开(公告)日：2009-01-15

申请号：PCT/US2008/008455

申请日：2008-07-10

Applicant: SANGAMO BIOSCIENCES, INC. ,  COLLINGWOOD, Trevor ,  COST, Gregory J.

Inventor： COLLINGWOOD, Trevor ,  COST, Gregory J.

IPC: C12N15/00 ,  C07K14/47 ,  C12N5/00 ,  C07K16/00

Abstract: Disclosed herein are methods and compositions for inactivating a FUT8 gene, using fusion proteins comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding said fusion proteins are also provided, as are cells comprising said polynucleotides and fusion proteins.