公开(公告)号：US20130171124A1

公开(公告)日：2013-07-04

申请号：US13557988

申请日：2012-07-25

申请人： Le Cong ,  Ann Marie Egloff ,  Levi A. Garraway ,  Jennifer Rubin Grandis ,  Eric S. Lander ,  Nicholas Stransky ,  Aaron D. Tward ,  Feng Zhang

发明人： Le Cong ,  Ann Marie Egloff ,  Levi A. Garraway ,  Jennifer Rubin Grandis ,  Eric S. Lander ,  Nicholas Stransky ,  Aaron D. Tward ,  Feng Zhang

IPC分类号： A61K38/16 ,  A61K45/06 ,  A61K38/46

CPC分类号： A61K38/164 ,  A61K38/465 ,  A61K45/06 ,  A61K48/005 ,  C07K14/4703 ,  C07K14/4705 ,  C07K2319/00 ,  C07K2319/70 ,  C07K2319/71 ,  C07K2319/80 ,  C12N9/22 ,  C12N15/907 ,  C12N2710/20011 ,  C12N2800/22 ,  C12N2999/007

摘要： The present invention provides methods related to the treatment of head and neck squamous cell carcinoma (HNSCC) and its associated premalignant lesions. In particular, the invention features methods which may specifically target HNSCC-associated genes and alter gene expression to treat or alleviate a symptom of HNSCC, or its related premalignant lesions. These methods may involve decreasing the function of an HNSCC-associated gene with aberrant gain-of-function; or increasing the function of an HNSCC-associated gene with aberrant loss-of-function.

摘要翻译： 本发明提供了与头颈鳞状细胞癌（HNSCC）及其相关的恶化前病变相关的方法。 特别地，本发明的特征在于可以特异性靶向HNSCC相关基因并改变基因表达以治疗或缓解HNSCC或其相关的恶化前病变的症状的方法。 这些方法可能涉及降低具有异常增殖功能的HNSCC相关基因的功能; 或增加具有异常功能丧失的HNSCC相关基因的功能。

公开(公告)号：US09370551B2

公开(公告)日：2016-06-21

申请号：US13557988

申请日：2012-07-25

申请人： Le Cong ,  Ann Marie Egloff ,  Levi A. Garraway ,  Jennifer Rubin Grandis ,  Eric S. Lander ,  Nicholas Stransky ,  Aaron D. Tward ,  Feng Zhang

发明人： Le Cong ,  Ann Marie Egloff ,  Levi A. Garraway ,  Jennifer Rubin Grandis ,  Eric S. Lander ,  Nicholas Stransky ,  Aaron D. Tward ,  Feng Zhang

IPC分类号： C12N15/85 ,  A61K38/16 ,  A61K38/46 ,  A61K45/06 ,  C07K14/47 ,  C12N9/22 ,  C12N15/90 ,  A61K48/00

CPC分类号： A61K38/164 ,  A61K38/465 ,  A61K45/06 ,  A61K48/005 ,  C07K14/4703 ,  C07K14/4705 ,  C07K2319/00 ,  C07K2319/70 ,  C07K2319/71 ,  C07K2319/80 ,  C12N9/22 ,  C12N15/907 ,  C12N2710/20011 ,  C12N2800/22 ,  C12N2999/007

摘要： The present invention provides methods related to the treatment of head and neck squamous cell carcinoma (HNSCC) and its associated premalignant lesions. In particular, the invention features methods which may specifically target HNSCC-associated genes and alter gene expression to treat or alleviate a symptom of HNSCC, or its related premalignant lesions. These methods may involve decreasing the function of an HNSCC-associated gene with aberrant gain-of-function; or increasing the function of an HNSCC-associated gene with aberrant loss-of-function.

摘要翻译： 本发明提供了与头颈鳞状细胞癌（HNSCC）及其相关的恶化前病变相关的方法。 特别地，本发明的特征在于可以特异性靶向HNSCC相关基因并改变基因表达以治疗或缓解HNSCC或其相关的恶化前病变的症状的方法。 这些方法可能涉及降低具有异常增殖功能的HNSCC相关基因的功能; 或增加具有异常功能丧失的HNSCC相关基因的功能。

公开(公告)号：US09499592B2

公开(公告)日：2016-11-22

申请号：US13353662

申请日：2012-01-19

申请人： Feng Zhang ,  Le Cong ,  Sriram Kosuri ,  George M. Church

发明人： Feng Zhang ,  Le Cong ,  Sriram Kosuri ,  George M. Church

IPC分类号： C07K14/195 ,  C07K14/47

CPC分类号： C07K14/195 ,  C07K14/4705 ,  C07K2319/00 ,  C07K2319/09 ,  C07K2319/60 ,  C07K2319/80 ,  C12P19/34

摘要： Provided herein are compositions, kits and methods useful in the construction of designer transcription activator-like effector (dTALE) polypeptides.

摘要翻译： 本文提供了可用于构建设计者转录激活子样效应子（dTALE）多肽的组合物，试剂盒和方法。

公开(公告)号：US20130137174A1

公开(公告)日：2013-05-30

申请号：US13604945

申请日：2012-09-06

申请人： Feng Zhang ,  Le Cong

发明人： Feng Zhang ,  Le Cong

IPC分类号： C12N5/071

CPC分类号： C12N5/06 ,  C07K14/03 ,  C07K14/195 ,  C07K14/4703 ,  C07K2319/80 ,  C12N5/0686 ,  C12N9/1241 ,  C12N9/22 ,  C12N15/635 ,  C12Q1/6816 ,  C12Q2522/10

摘要： The invention relates to methods of altering expression of a genomic locus of interest or specifically targeting a genomic locus of interest in an animal cell, which may involve contacting the genomic locus with a non-naturally occurring or engineered composition that includes a deoxyribonucleic acid (DNA) binding polypeptide having a N-terminal capping region, a DNA binding domain comprising at least five or more Transcription activator-like effector (TALE) monomers and at least one or more half-monomers specifically ordered to target the genomic locus of interest, and a C-terminal capping region, wherein the polypeptide includes at least one or more effector domains, and wherein the polypeptide is encoded by and translated from a codon optimized nucleic acid molecule so that the polypeptide preferentially binds to the DNA of the genomic locus.

公开(公告)号：US20140186958A1

公开(公告)日：2014-07-03

申请号：US14105017

申请日：2013-12-12

申请人： Feng Zhang ,  Le Cong ,  Randall Jeffrey Platt ,  Neville Espi Sanjana ,  Fei Ran

发明人： Feng Zhang ,  Le Cong ,  Randall Jeffrey Platt ,  Neville Espi Sanjana ,  Fei Ran

IPC分类号： C12N15/85

CPC分类号： C12N15/63 ,  C12N7/00 ,  C12N9/22 ,  C12N15/01 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/85 ,  C12N15/86 ,  C12N2320/30 ,  C12N2740/15043

摘要： The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors with additional functional domains. Also provided are methods of directing CRISPR complex formation in prokaryotic and eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity.

公开(公告)号：US20130137173A1

公开(公告)日：2013-05-30

申请号：US13554922

申请日：2012-07-20

申请人： Feng Zhang ,  Le Cong

发明人： Feng Zhang ,  Le Cong

IPC分类号： C12N5/071

CPC分类号： C12N15/63

摘要： The invention relates to methods of altering expression of a genomic locus of interest or specifically targeting a genomic locus of interest in an animal cell, which may involve contacting the genomic locus with a non-naturally occurring or engineered composition that includes a deoxyribonucleic acid (DNA) binding polypeptide having a N-terminal capping region, a DNA binding domain comprising at least five or more Transcription activator-like effector (TALE) monomers and at least one or more half-monomers specifically ordered to target the genomic locus of interest, and a C-terminal capping region, wherein the polypeptide includes at least one or more effector domains, and wherein the polypeptide is encoded by and translated from a codon optimized nucleic acid molecule so that the polypeptide preferentially binds to the DNA of the genomic locus.

摘要翻译： 本发明涉及改变感兴趣的基因座的表达或特异性靶向动物细胞中的目的基因组的表达的方法，其可能涉及使基因组座位与包含脱氧核糖核酸（DNA）的非天然存在或工程化组合物接触 ）结合多肽，DNA结合结构域包含至少五个或更多个转录激活子样效应子（TALE）单体和至少一种或多种特异性命令靶向感兴趣的基因组座位的半单体，以及 C端封端区，其中所述多肽包括至少一个或多个效应域，并且其中所述多肽由密码子优化的核酸分子编码和翻译，使得所述多肽优先结合所述基因组座位的DNA。

公开(公告)号：US08450107B1

公开(公告)日：2013-05-28

申请号：US13604945

申请日：2012-09-06

申请人： Feng Zhang ,  Le Cong

发明人： Feng Zhang ,  Le Cong

IPC分类号： C12N15/85 ,  C12N15/00

CPC分类号： C12N5/06 ,  C07K14/03 ,  C07K14/195 ,  C07K14/4703 ,  C07K2319/80 ,  C12N5/0686 ,  C12N9/1241 ,  C12N9/22 ,  C12N15/635 ,  C12Q1/6816 ,  C12Q2522/10

摘要： The invention relates to methods of altering expression of a genomic locus of interest or specifically targeting a genomic locus of interest in an animal cell, which may involve contacting the genomic locus with a non-naturally occurring or engineered composition that includes a deoxyribonucleic acid (DNA) binding polypeptide having a N-terminal capping region, a DNA binding domain comprising at least five or more Transcription activator-like effector (TALE) monomers and at least one or more half-monomers specifically ordered to target the genomic locus of interest, and a C-terminal capping region, wherein the polypeptide includes at least one or more effector domains, and wherein the polypeptide is encoded by and translated from a codon optimized nucleic acid molecule so that the polypeptide preferentially binds to the DNA of the genomic locus.

摘要翻译： 本发明涉及改变感兴趣的基因座的表达或特异性靶向动物细胞中的目的基因组的表达的方法，其可能涉及使基因组座位与包含脱氧核糖核酸（DNA）的非天然存在或工程化组合物接触 ）结合多肽，DNA结合结构域包含至少五个或更多个转录激活子样效应子（TALE）单体和至少一种或多种特异性命令靶向感兴趣的基因组座位的半单体，以及 C端封端区，其中所述多肽包括至少一个或多个效应域，并且其中所述多肽由密码子优化的核酸分子编码和翻译，使得所述多肽优先结合所述基因组座位的DNA。

公开(公告)号：US20120270273A1

公开(公告)日：2012-10-25

申请号：US13353662

申请日：2012-01-19

申请人： Feng Zhang ,  Le Cong ,  Sriram Kosuri ,  George M. Church

发明人： Feng Zhang ,  Le Cong ,  Sriram Kosuri ,  George M. Church

IPC分类号： C12N15/62 ,  C12P19/34 ,  C12N15/63

CPC分类号： C07K14/195 ,  C07K14/4705 ,  C07K2319/00 ,  C07K2319/09 ,  C07K2319/60 ,  C07K2319/80 ,  C12P19/34

摘要： Provided herein are compositions, kits and methods useful in the construction of designer transcription activator-like effector (dTALE) polypeptides.

摘要翻译： 本文提供了可用于构建设计者转录激活子样效应子（dTALE）多肽的组合物，试剂盒和方法。