专利快速检索-快速检索全球专利，免费商用专利数据库-IPRDB

1. 发明申请

WO2018053053A1 PROXIMITY-DEPENDENT BIOTINYLATION AND USES THEREOF 审中-公开
标题翻译：接近邻苯二甲酸生物化及其用途
公开(公告)号：WO2018053053A1
公开(公告)日：2018-03-22
申请号：PCT/US2017/051428
申请日：2017-09-13
申请人： THE BROAD INSTITUTE, INC. , CARR, Steven, A. , WRIGHT, Jason , MYERS, Sam
发明人： CARR, Steven, A. , WRIGHT, Jason , MYERS, Sam
IPC分类号： A61K48/00 , C12N9/22 , C12N15/63 , C12N15/79 , G06F19/16
CPC分类号： C12N9/22 , C07K2319/09 , C07K2319/85 , C12N9/0065 , C12N9/93 , C12N15/102 , C12N15/11 , C12N15/63 , C12N2310/20 , C12N2740/16043 , C12Y111/01011 , C12Y603/04015 , G16B20/00
摘要： The present invention provides fusion proteins, polynucleotides, kits, as well as TALE- or CRISPR-Cas based systems and methods. The present invention relies on proximity-dependent biotinylation, which allows site-directed protein or DNA purification and identification. The present invention provides tools for delineating the genetics of disease mechanism and for the identification of therapeutic targets and markers.
摘要翻译：本发明提供了融合蛋白，多核苷酸，试剂盒以及基于TALE或CRISPR-Cas的系统和方法。本发明依赖邻近依赖性生物素化，其允许定点蛋白质或DNA纯化和鉴定。本发明提供了用于划定疾病机制的遗传学以及用于鉴定治疗靶标和标记的工具。

2. 发明申请

WO2017070633A2 EVOLVED CAS9 PROTEINS FOR GENE EDITING 审中-公开
标题翻译：用于基因编辑的演化的CAS9蛋白
公开(公告)号：WO2017070633A2
公开(公告)日：2017-04-27
申请号：PCT/US2016/058345
申请日：2016-10-22
申请人： PRESIDENT AND FELLOWS OF HARVARD COLLEGE , LIU, David, R. , HU, Johnny, Hao
发明人： LIU, David, R. , HU, Johnny, Hao
IPC分类号： C07K14/32
CPC分类号： C12N9/22 , C07K14/32 , C07K2319/00 , C07K2319/09 , C07K2319/80 , C12N9/2497 , C12N9/78 , C12N15/907 , C12Y305/04005
摘要： Some aspects of this disclosure provide strategies, systems, reagents, methods, and kits that are useful for enginerring Cas9 and Cas9 variants that have increased activity on target sequences that do not contain the canonical PAM sequence. In some embodiments, fusion proteins comprising such Cas9 variants and nucleic acid editing domains, e.g., deaminase domains, are provided.
摘要翻译：本公开的一些方面提供了对不具有经典PAM序列的靶序列具有增加的活性的工程改造Cas9和Cas9变体有用的策略，系统，试剂，方法和试剂盒。在一些实施方案中，提供了包含此类Cas9变体和核酸编辑结构域例如脱氨酶结构域的融合蛋白。

3. 发明申请

WO2017070029A1 METHODS AND COMPOSITIONS FOR MARKER-FREE GENOME MODIFICATION 审中-公开
标题翻译：无标记基因组修饰的方法和组合物
公开(公告)号：WO2017070029A1
公开(公告)日：2017-04-27
申请号：PCT/US2016/057272
申请日：2016-10-17
申请人： PIONEER HI-BRED INTERNATIONAL, INC.
发明人： CIGAN, Andrew Mark , SVITASHEV, Sergei
IPC分类号： C12N9/22 , C12N15/01 , C12N15/82 , C12N15/90
CPC分类号： C12N15/8206 , C07K2319/09 , C12N9/22 , C12N15/8207 , C12N15/8241 , C12N15/902
摘要： Compositions and methods are provided for modifying a nucleotide sequence in the genome of a plant cell, without the use of a selectable marker. The methods and compositions employ a guide polynucleotide/Cas endonuclease system to make a double strand break in a target site located in a nucleotide sequence and plant cells are obtained without the use of a selectable marker, and to provide an effective system for modifying target sites within the genome of a plant, plant cell or seed. Compositions and methods are also provided for producing a plant cell, callus tissue or plant having a modified nucleotide sequence in its genome, without the use of a selectable marker.
摘要翻译：提供组合物和方法用于修饰植物细胞基因组中的核苷酸序列，而不使用选择标记。所述方法和组合物使用引导多核苷酸/ Cas核酸内切酶系统在位于核苷酸序列中的靶位点中产生双链断裂，并且在不使用选择标记的情况下获得植物细胞，并提供用于修饰靶位点的有效系统在植物，植物细胞或种子的基因组内。还提供了组合物和方法，用于在其基因组中产生具有修饰的核苷酸序列的植物细胞，愈伤组织或植物，而不使用选择标记。

4. 发明申请

WO2016205759A1 ENGINEERING AND OPTIMIZATION OF SYSTEMS, METHODS, ENZYMES AND GUIDE SCAFFOLDS OF CAS9 ORTHOLOGS AND VARIANTS FOR SEQUENCE MANIPULATION 审中-公开
标题翻译： CAS9 ORTHOLOGS的系统，方法，酶和指导原则的工程与优化及其对序列操作的变体
公开(公告)号：WO2016205759A1
公开(公告)日：2016-12-22
申请号：PCT/US2016/038252
申请日：2016-06-17
申请人： THE BROAD INSTITUTE INC. , MASSACHUSETTS INSTITUTE OF TECHNOLOGY , PRESIDENT AND FELLOWS OF HARVARD COLLEGE , UNIVERSITY OF TOKYO , ZHANG, Feng , YAN, Winston , NUREKI, Osamu , ZHENG, Kaijie , CONG, Le , NISHIMASU, Hiroshi , RAN, Fei , LI, Yinqing
发明人： ZHANG, Feng , YAN, Winston , NUREKI, Osamu , ZHENG, Kaijie , CONG, Le , NISHIMASU, Hiroshi , RAN, Fei , LI, Yinqing
IPC分类号： C12N9/22 , G06F19/16 , A61K48/00 , C12N15/63 , C12N15/79
CPC分类号： C12N9/22 , C07K2299/00 , C07K2319/09 , C07K2319/095 , C07K2319/20 , C07K2319/70 , C12N15/102
摘要： The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems. In particular the present invention comprehends engineered new guide architectures and enzymes to be used in optimized Staphylococcus aureus CRISPR-Cas enzyme systems.
摘要翻译：本发明提供用于改变靶基因序列和相关基因产物的表达的系统，方法和组合物。提供了关于CRISPR-Cas系统的Cas蛋白的结构信息，使用该信息来生成CRISPR复合物的修饰组分，编码CRISPR复合物的一种或多种组分或修饰组分的载体和载体系统，以及方法用于设计和使用这些矢量和组件。还提供了在真核细胞中引导CRISPR复合物形成的方法以及利用CRISPR-Cas系统的方法。特别地，本发明包括优化的功能性CRISPR-Cas酶系统。特别地，本发明包括用于优化的金黄色葡萄球菌CRISPR-Cas酶系统中的工程新指南体系结构和酶。

5. 发明申请

WO2016170484A1 RNA-GUIDED GENE EDITING SYSTEM AND USES THEREOF 审中-公开
标题翻译： RNA指导基因编码系统及其用途
公开(公告)号：WO2016170484A1
公开(公告)日：2016-10-27
申请号：PCT/IB2016/052243
申请日：2016-04-20
申请人： NOVARTIS AG , LOEW, Andreas , HUEBNER, Anette
发明人： LOEW, Andreas , HUEBNER, Anette
IPC分类号： C12N15/90 , C12N15/10 , C12N15/63 , C12N5/00
CPC分类号： C12N9/22 , A61K38/00 , A61K48/00 , C07K2319/09 , C07K2319/21 , C07K2319/85 , C12N9/2462 , C12N15/102 , C12N15/113 , C12N15/115 , C12N15/63 , C12N2310/11 , C12N2310/16 , C12N2310/3519 , C12N2320/34 , C12N2800/80
摘要： The present invention provides RNA-guided gene editing systems and methods of use thereof.
摘要翻译：本发明提供了RNA引导的基因编辑系统及其使用方法。

6. 发明申请

WO2016098078A3 DIMERIZATION SWITCHES AND USES THEREOF 审中-公开
标题翻译：二化化开关及其用途
公开(公告)号：WO2016098078A3
公开(公告)日：2016-10-27
申请号：PCT/IB2015059796
申请日：2015-12-18
申请人： NOVARTIS AG , LOEW ANDREAS , VASH BRIAN
发明人： LOEW ANDREAS , VASH BRIAN
IPC分类号： C12N15/62 , C12N9/12 , C12N9/90
CPC分类号： C12N9/90 , C07K2319/09 , C07K2319/70 , C07K2319/81 , C12N9/12 , C12Y207/11001 , C12Y502/01008
摘要： The present invention provides gene editing systems comprising gene editing dimerization switches comprising a first and second gene editing switch domain that allow for the regulation of a gene editing function by the introduction, e.g., administration, of a gene editing dimerization molecule having the ability to bring together a first gene editing switch domain and a second gene editing switch domain. A regulated gene editing function provides, e.g., less off-target side effects, and increases the therapeutic window. The present invention also provides improved FKBP/FRB-based dimerization switches wherein the FRB switch domain or the FKBP switch domain, or both the FRB and FKBP switch domains, comprise one or more mutations that optimize performance, e.g., that alter, e.g., enhance the formation of a complex between the first switch domain, the second switch domain, and the dimerization molecule, rapamycin, or a rapalog, e.g., RAD001.
摘要翻译：本发明提供了基因编辑系统，其包含基因编辑二聚化开关，所述基因编辑二聚化开关包含第一和第二基因编辑开关结构域，所述第一和第二基因编辑开关结构域允许通过引入（例如施用）编码二聚化分子的基因来调节基因编辑功能，一起构成第一基因编辑开关域和第二基因编辑开关域。受管理的基因编辑功能提供了例如更少的脱靶副作用，并增加了治疗窗口。本发明还提供了改进的基于FKBP / FRB的二聚化开关，其中FRB开关结构域或FKBP开关结构域或FRB和FKBP开关结构域两者包含一个或多个优化性能的突变，例如改变例如增强第一开关结构域，第二开关结构域和二聚化分子雷帕霉素或雷帕霉素例如RAD001之间形成复合物。

7. 发明申请

WO2016161004A1 COMPOSITIONS COMPRISING TALENS AND METHODS OF TREATING HIV 审中-公开
标题翻译：包含孕妇和治疗艾滋病毒的方法的组合物
公开(公告)号：WO2016161004A1
公开(公告)日：2016-10-06
申请号：PCT/US2016/025037
申请日：2016-03-30
申请人： THE BOARD OF REGENTS OF THE NEVADA SYSTEM OF HIGHER EDUC. ON BEHALF OF THE UNIVERSITY OF NEVADA, LA
发明人： SCHILLER, Martin, R. , STRONG, Christy, L.
IPC分类号： C12N9/16 , C12N15/00 , C12N5/07 , C12P19/34 , A61K38/00
CPC分类号： C12N15/1132 , A61K31/7088 , A61K31/7105 , A61K38/03 , A61K45/06 , A61K48/0066 , A61K48/0075 , A61P31/18 , C07K2319/09 , C07K2319/80 , C12N9/22 , C12P19/34 , C12Y301/21004
摘要： Disclosed herein, are compositions and methods for the treatment of human immunodeficiency virus infection. The compositions comprise engineered transcription activator like effector nucleases (TALENs) comprising a TALE DNA binding domain flanked by two spacer sequences, and a Fokl nuclease catalytic domain. Also, described herein, are methods of using TALENs to cleave nucleic acids; and methods of administering the TALENs to subjects at risk for or having an HIV infection.
摘要翻译：本文公开了用于治疗人类免疫缺陷病毒感染的组合物和方法。组合物包含工程转录激活剂如效应核酸酶（TALEN），其包含侧翼为两个间隔序列的TALE DNA结合结构域和Fok1核酸酶催化结构域。此外，本文描述的是使用TALEN切割核酸的方法; 以及将TALEN施用于患有或具有HIV感染风险的受试者的方法。

8. 发明申请

WO2016094872A9 DEAD GUIDES FOR CRISPR TRANSCRIPTION FACTORS 审中-公开
标题翻译： CRISPR转录因子的死亡指南
公开(公告)号：WO2016094872A9
公开(公告)日：2016-08-18
申请号：PCT/US2015065393
申请日：2015-12-11
申请人： BROAD INST INC , MASSACHUSETTS INST TECHNOLOGY
发明人： ZHANG FENG , KONERMANN SILVANA , DAHLMAN JAMES , ABUDAYYEH OMAR O
IPC分类号： C12N15/11
CPC分类号： C12N15/113 , A01K67/0275 , A01K2207/12 , A01K2217/05 , A01K2227/105 , A01K2267/03 , A61K48/005 , C07K2319/00 , C07K2319/09 , C12N9/22 , C12N15/111 , C12N15/115 , C12N15/635 , C12N2310/16 , C12N2310/20 , C12N2310/3519 , C12Q1/6876 , C12Q2600/178 , C12Y301/21004
摘要： The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems.
摘要翻译：本发明提供用于改变靶基因序列和相关基因产物的表达的系统，方法和组合物。提供了关于CRISPR-Cas系统的Cas蛋白的结构信息，使用该信息来生成CRISPR复合物的修饰组分，编码CRISPR复合物的一种或多种组分或修饰组分的载体和载体系统，以及方法用于设计和使用这些矢量和组件。还提供了在真核细胞中引导CRISPR复合物形成的方法以及利用CRISPR-Cas系统的方法。特别地，本发明包括优化的功能性CRISPR-Cas酶系统。

9. 发明申请

WO2016094872A1 DEAD GUIDES FOR CRISPR TRANSCRIPTION FACTORS 审中-公开
标题翻译： CRISPR转录因子死亡指南
公开(公告)号：WO2016094872A1
公开(公告)日：2016-06-16
申请号：PCT/US2015/065393
申请日：2015-12-11
申请人： THE BROAD INSTITUTE INC. , MASSACHUSETTS INSTITUTE OF TECHNOLOGY
发明人： ZHANG, Feng , KONERMANN, Silvana , DAHLMAN, James , ABUDAYYEH, Omar, O.
IPC分类号： C12N15/11
CPC分类号： C12N15/113 , A01K67/0275 , A01K2207/12 , A01K2217/05 , A01K2227/105 , A01K2267/03 , A61K48/005 , C07K2319/00 , C07K2319/09 , C12N9/22 , C12N15/111 , C12N15/115 , C12N15/635 , C12N2310/16 , C12N2310/20 , C12N2310/3519 , C12Q1/6876 , C12Q2600/178 , C12Y301/21004
摘要： The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems.
摘要翻译：本发明提供了用于改变靶基因序列和相关基因产物表达的系统，方法和组合物。提供了关于CRISPR-Cas系统的Cas蛋白的结构信息，使用该信息产生CRISPR复合物的修饰组分，编码CRISPR复合物的一种或多种组分或修饰组分的载体和载体系统，以及方法用于这些载体和组件的设计和使用。还提供了指导真核细胞中CRISPR复合物形成的方法和利用CRISPR-Cas系统的方法。特别地，本发明包含优化的功能性CRISPR-Cas酶系统。

10. 发明申请

WO2016073433A1 PEPTIDE-MEDIATED DELIVERY OF RNA-GUIDED ENDONUCLEASE INTO CELLS 审中-公开
标题翻译：肽介导的RNA引导的内切酶进入细胞
公开(公告)号：WO2016073433A1
公开(公告)日：2016-05-12
申请号：PCT/US2015/058760
申请日：2015-11-03
申请人： E. I. DU PONT DE NEMOURS AND COMPANY
发明人： FRISCH, Ryan L. , FAN, Xiaochun , HONG, Seung-Pyo , JACKSON, Ethel Noland
IPC分类号： C12N9/22 , C12N15/62
CPC分类号： C12N9/22 , C07K2319/09 , C07K2319/10 , C12N9/96 , C12N15/102 , C12N15/62 , C12N15/86 , C12N15/907 , C12N2310/3513 , C12N2750/14141
摘要： A composition is disclosed that comprises at least one protein component of an RNA-guided endonuclease (RGEN) and at least one cell-penetrating peptide (CPP), wherein the RGEN protein component and CPP are covalently or non-covalently linked to each other in an RGEN protein-CPP complex. The RGEN protein-CPP complex can traverse (i) a cell membrane, or (ii) a cell wall and cell membrane, of a cell. The RGEN protein component of an RGEN protein-CPP complex in certain embodiments can be associated with a suitable RNA component to provide an RGEN capable of specific DNA targeting. Further disclosed are compositions comprising at least one protein component of a guide polynucleotide/Cas endonuclease complex and at least one CPP, as well as methods of delivering RGEN proteins into microbial cells, as well as methods of targeting DNA with RGENs.
摘要翻译：公开了包含RNA引导的内切核酸酶（RGEN）和至少一种细胞穿透肽（CPP）的至少一种蛋白质组分的组合物，其中所述RGEN蛋白质组分和CPP彼此共价或非共价连接 RGEN蛋白-CPP复合物。 RGEN蛋白-CPP复合物可以穿过（i）细胞膜，或（ii）细胞的细胞壁和细胞膜。在一些实施方案中，RGEN蛋白-CPP复合物的RGEN蛋白组分可以与合适的RNA组分相关联，以提供能够进行特异性DNA靶向的RGEN。还公开了包含导向多核苷酸/ Cas内切核酸酶复合物和至少一种CPP的至少一种蛋白质组分的组合物，以及将RGEN蛋白递送到微生物细胞中的方法，以及用RGEN靶向DNA的方法。

你已经成功收藏专利！

检索式保存成功!

IPRDB

热门服务

关于我们

友情链接

联系方式