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1. 发明申请

WO2005102038A3 EXPRESSION OF POLYPEPTIDES IN ROD OUTER SEGMENT MEMBRANES 审中-公开
标题翻译：多糖分子膜中多聚体的表达
公开(公告)号：WO2005102038A3
公开(公告)日：2006-07-27
申请号：PCT/US2005013742
申请日：2005-04-22
申请人： NOVASITE PHARMACEUTICALS INC , PALCZEWSKI KRZYSZTOF , LI NING , BALLESTEROS JUAN
发明人： PALCZEWSKI KRZYSZTOF , LI NING , BALLESTEROS JUAN
IPC分类号： C12N15/00 , A01H5/00 , A01K67/00 , A01K67/027 , A61K48/00 , C07K14/47 , C07K14/705 , C07K14/72 , C07K19/00 , C12N5/10 , C12N15/09 , C12N15/63 , C12N15/85 , C12P21/00 , G01N33/00
CPC分类号： C07K14/723 , A01K67/0275 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2227/105 , A01K2227/50 , A01K2267/01 , A01K2267/03 , C07K14/705 , C07K2319/01 , C07K2319/40 , C12N15/8509 , C12N2800/30 , C12N2810/00 , C12N2830/008
摘要： The invention provides a transgene construct consisting of a transgene encoding a membrane polypeptide comprising a rod outer segment (ROS) targeting signal, and an operably associated species specific rod-specific regulatory sequence, wherein said polypeptide is not rhodopsin. The membrane polypeptide can be a G protein-coupled receptor (GPCR). Transgenic membrane polypeptides include a receptor selected from 5HT 1A , 5HT 1B , 5HT 1D , 5HT 1E , 5HT 1F , 5HT 2A , 5HT 2B , 5HT 2C , 5HT 4A , 5HT 5A , 5HT 6 , 5HT 7A , EDG1, EDG2, EDG3, EDG4, EDG5, EDG6, EDG7, EDG8, CB2, FMLP and MC4. The invention also provides a Xenopus cell whose genome contains a transgene encoding a membrane polypeptide comprising a ROS targeting signal operably associated with a Xenopus rod-specific regulatory sequence, wherein said polypeptide is not a rhodopsin. A Xenopus tadpole or adult whose genome contains a transgene encoding a membrane polypeptide comprising a ROS targeting signal operably associated with a Xenopus rod-specific regulatory sequence, wherein said polypeptide is not a rhodopsin is further provided.
摘要翻译：本发明提供由编码包含杆外节段（ROS）靶向信号的膜多肽的转基因和可操作地相关的物种特异性杆特异性调节序列组成的转基因构建体，其中所述多肽不是视紫红质。膜多肽可以是G蛋白偶联受体（GPCR）。转基因膜多肽包括选自5HT 1A，5HT 1B，5HT 1D，5HT 1E，5HT， 5HT 2A，5HT 2B，5HT 2C，5HT 4A，5HT 2，5HT 2， 5HT 6，5HT 7A，EDG1，EDG2，EDG3，EDG4，EDG5，EDG6，EDG7，EDG8，CB2，FMLP和MC4。本发明还提供了一种非洲爪蟾细胞，其基因组含有编码膜多肽的转基因，所述多肽包含与非洲爪蟾杆特异性调节序列可操作地相关的ROS靶向信号，其中所述多肽不是视紫红质。非洲爪蟾ole属或成年人，其基因组含有编码膜多肽的转基因，所述多肽包含与非洲爪蟾杆特异性调节序列可操作地相关的ROS靶向信号，其中所述多肽不是视紫红质。

2. 发明申请

WO2005102038A2 EXPRESSION OF POLYPEPTIDES IN ROD OUTER SEGMENT MEMBRANES 审中-公开
标题翻译：多糖分子膜中多聚体的表达
公开(公告)号：WO2005102038A2
公开(公告)日：2005-11-03
申请号：PCT/US2005/013742
申请日：2005-04-21
申请人： NOVASITE PHARMACEUTICALS, INC. , PALCZEWSKI, Krzysztof , LI, Ning , BALLESTEROS, Juan
发明人： PALCZEWSKI, Krzysztof , LI, Ning , BALLESTEROS, Juan
IPC分类号： A01K67/00
CPC分类号： C07K14/723 , A01K67/0275 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2227/105 , A01K2227/50 , A01K2267/01 , A01K2267/03 , C07K14/705 , C07K2319/01 , C07K2319/40 , C12N15/8509 , C12N2800/30 , C12N2810/00 , C12N2830/008
摘要： The invention provides a transgene construct consisting of a transgene encoding a membrane polypeptide comprising a rod outer segment (ROS) targeting signal, and an operably associated species specific rod-specific regulatory sequence, wherein said polypeptide is not rhodopsin. The membrane polypeptide can be a G protein-coupled receptor (GPCR). Transgenic membrane polypeptides include a receptor selected from 5HT 1A , 5HT 1B , 5HT 1D , 5HT 1E , 5HT 1F , 5HT 2A , 5HT 2B , 5HT 2C , 5HT 4A , 5HT 5A , 5HT 6 , 5HT 7A , EDG1, EDG2, EDG3, EDG4, EDG5, EDG6, EDG7, EDG8, CB2, FMLP and MC4. The invention also provides a Xenopus cell whose genome contains a transgene encoding a membrane polypeptide comprising a ROS targeting signal operably associated with a Xenopus rod-specific regulatory sequence, wherein said polypeptide is not a rhodopsin. A Xenopus tadpole or adult whose genome contains a transgene encoding a membrane polypeptide comprising a ROS targeting signal operably associated with a Xenopus rod-specific regulatory sequence, wherein said polypeptide is not a rhodopsin is further provided.
摘要翻译：本发明提供由编码包含杆外节段（ROS）靶向信号的膜多肽的转基因和可操作地相关的物种特异性杆特异性调节序列组成的转基因构建体，其中所述多肽不是视紫红质。膜多肽可以是G蛋白偶联受体（GPCR）。转基因膜多肽包括选自5HT1A，5HT1B，5HT1D，5HT1E，5HT1F，5HT2A，5HT2B，5HT2C，5HT4A，5HT5A，5HT6,5HT7A，EDG1，EDG2，EDG3，EDG4，EDG5，EDG6，EDG7，EDG8，CB2， FMLP和MC4。本发明还提供了一种非洲爪蟾细胞，其基因组含有编码膜多肽的转基因，所述多肽包含与非洲爪蟾杆特异性调节序列可操作地相关的ROS靶向信号，其中所述多肽不是视紫红质。非洲爪蟾ole属或成年人，其基因组含有编码膜多肽的转基因，所述多肽包含与非洲爪蟾杆特异性调节序列可操作地相关的ROS靶向信号，其中所述多肽不是视紫红质。

3. 发明申请

WO2005024382A2 NANODEVICES EMPLOYING COMBINATORIAL ARTIFICIAL RECEPTORS 审中-公开
公开(公告)号：WO2005024382A2
公开(公告)日：2005-03-17
申请号：PCT/US2004029046
申请日：2004-09-03
申请人： RECEPTORS LLC , CARLSON ROBERT E
发明人： CARLSON ROBERT E
IPC分类号： B01J19/00 , G01N33/543 , G01N33/566 , G01N33/68 , G01N
CPC分类号： G01N33/588 , B01J19/0046 , B01J2219/00524 , B01J2219/00527 , B01J2219/00576 , B01J2219/00605 , B01J2219/00612 , B01J2219/00626 , B01J2219/00659 , B01J2219/00702 , B01J2219/00722 , B01J2219/00725 , B01J2219/00731 , B01J2219/00738 , B82Y5/00 , B82Y10/00 , B82Y15/00 , B82Y30/00 , C07K14/705 , C12N2810/00 , C12N2810/50 , C40B80/00 , G01N33/54326 , G01N33/54346 , G01N33/552
摘要： The present invention includes nanodevices employing combinatorial artificial receptors and methods for making and using the same. In an embodiment the invention includes a method of adhering components together. In an embodiment, the invention includes a device including a first component adhered to a second component via a binding pair of artificial receptors. In an embodiment, the invention includes an agent delivery device having a capsule, and an active agent. In an embodiment, the invention can include a detection device having a magnetic particle and an artificial receptor disposed thereon. In an embodiment, the invention can include a detection device having a quantum dot and an artificial receptor disposed on the quantum dot. In an embodiment, the invention includes a detection device having first particles and second particles that aggregate in the present of a target ligand. In an embodiment, the invention includes a detection device having a cantilever and an artificial receptor disposed thereon. In an embodiment, the invention can include a detection device having a substrate and an artificial receptor disposed thereon. In an embodiment, the invention can include a device for selective removal of a target component including a substrate and an artificial receptor disposed thereon.

4. 发明申请

WO01088160A2 VECTORS FOR DNA DELIVERY 审中-公开
标题翻译： DNA交付的向量
公开(公告)号：WO01088160A2
公开(公告)日：2001-11-22
申请号：PCT/IB2001/000980
申请日：2001-05-11
IPC分类号： A61K48/00 , C07B61/00 , C07D233/90 , C07K1/04 , C07K7/06 , C07K7/08 , C12N15/63
CPC分类号： C40B50/14 , A61K48/00 , C07B2200/11 , C07K1/04 , C07K7/06 , C07K7/08 , C12N15/87 , C12N2810/00
摘要： The present invention pertains to novel products suitable for use as gene delivery systems in which nucleic acid is linked to a ligand in order to facilitate delivery of the nucleic acid to a target cell or sub-cellular compartment via uptake of the ligand. More particularly, the present invention pertains to vectors comprising: (a) a double stranded DNA (dsDNA) having at least one target sequence; and, (b) a chimeric molecule comprising: (i) a sequence specific polyamide (SSP) moiety bound non-covalently to said target sequence; and, (ii) a ligand moiety linked covalently to said sequence specific polyamide. The present invention also pertains to compositions comprising such chimeric molecules and vectors; methods for making such chimeric molecules and vectors; and methods of using such chimeric molecules and vectors, e.g., to deliver nucleic acid vectors to cells or sub-cellular compartments.
摘要翻译：本发明涉及适合用作基因递送系统的新产品，其中核酸与配体连接以便于通过摄取配体来促进将核酸递送至靶细胞或亚细胞区室。更具体地，本发明涉及包含以下的载体：（a）具有至少一个靶序列的双链DNA（dsDNA）; 和（b）嵌合分子，其包含：（i）与所述靶序列非共价结合的序列特异性聚酰胺（SSP）部分; 和（ii）与所述序列特异性聚酰胺共价连接的配体部分。本发明还涉及包含这种嵌合分子和载体的组合物; 制备这种嵌合分子和载体的方法; 以及使用这种嵌合分子和载体的方法，例如将核酸载体递送至细胞或亚细胞区室。

5. 发明申请

WO2017077275A1 PHAGEMID VECTOR 审中-公开
标题翻译： PHAGEMID矢量
公开(公告)号：WO2017077275A1
公开(公告)日：2017-05-11
申请号：PCT/GB2016/053366
申请日：2016-10-31
申请人： IMPERIAL INNOVATIONS LIMITED
发明人： HAJITOU, Amin , ASAVARUT, Paladd , YATA, Teerapong
IPC分类号： C12N15/86 , A61K48/00 , A61K39/00
CPC分类号： C12N15/86 , A61K31/495 , A61K39/00 , A61K48/00 , A61K2039/5256 , C12N2750/14134 , C12N2750/14143 , C12N2750/14144 , C12N2750/14152 , C12N2795/00043 , C12N2795/00044 , C12N2795/00045 , C12N2795/14134 , C12N2795/14143 , C12N2795/14144 , C12N2795/14152 , C12N2799/021 , C12N2799/04 , C12N2799/06 , C12N2810/00 , C12N2810/405 , C12N2820/10 , C12N2820/55 , C12N2820/60
摘要： The invention provides hybrid and recombinant phagemid vectors for expressing a transgene in a target cell transduced with the vector. A recombinant phagemid particle comprises at least one transgene expression cassette which encodes an agent which exerts a biological effect on the target cell, characterised in that the phagemid particle comprises a genome which lacks at least 50% of its bacteriophage genome. The invention extends to the use of such phagemid expression systems as a research tool, and for the delivery of transgenes in a variety of gene therapy applications, DNA and/or peptide vaccine delivery and imaging techniques. The invention extends to in vitro, in vivo or in situ methods for producing viral vectors, such as recombinant adeno- associated viruses (rAAV) or lentivirus vectors (rLV), and to genetic constructs used in such methods.
摘要翻译：本发明提供了用于在用该载体转导的靶细胞中表达转基因的杂交和重组噬菌粒载体。重组噬菌粒粒子包含至少一个转基因表达盒，其编码对靶细胞发挥生物学作用的试剂，其特征在于所述噬菌粒粒子包含缺少其50％噬菌体基因组的基因组。本发明扩展到使用这种噬菌粒表达系统作为研究工具，并且用于在各种基因治疗应用中递送转基因，DNA和/或肽疫苗递送和成像技术。本发明延伸至用于生产病毒载体（例如重组腺相关病毒（rAAV）或慢病毒载体（rLV））的体外，体内或原位方法，以及用于此类方法中的遗传构建体。

6. 发明申请

WO2012027358A1 OPTOGENETIC PROBES FOR MEASURING MEMBRANE POTENTIAL 审中-公开
标题翻译：用于测量膜电位的原子探针
公开(公告)号：WO2012027358A1
公开(公告)日：2012-03-01
申请号：PCT/US2011/048793
申请日：2011-08-23
申请人： PRESIDENT AND FELLOWS OF HARVARD COLLEGE , COHEN, Adam E. , KRALJ, Joel M. , DOUGLASS, Adam D.
发明人： COHEN, Adam E. , KRALJ, Joel M. , DOUGLASS, Adam D.
IPC分类号： G01N33/50 , G01N33/68
CPC分类号： G01N33/6872 , C07K14/00 , C07K14/195 , C07K14/43595 , C07K2319/60 , C12N7/00 , C12N15/62 , C12N15/625 , C12N15/85 , C12N15/86 , C12N2740/15043 , C12N2750/14143 , C12N2810/00 , G01N21/6486 , G01N33/566 , G01N2333/726
摘要： The invention provides methods, cells and constructs for optical measurement of membrane potential. These methods can be used in cells that are not accessible to presently available methods using electrodes. The methods can be directed to, for example, high-throughput drug screening assays to determine agents that can affect membrane potential of a target cell.
摘要翻译：本发明提供了用于光学测量膜电位的方法，细胞和构建体。这些方法可以用于目前可用的使用电极的方法不可及的细胞中。该方法可以针对例如高通量药物筛选测定来确定可影响靶细胞膜电位的试剂。

7. 发明申请

WO2007089632A2 HEPARIN AND HEPARAN SULFATE BINDING CHIMERIC VECTORS 审中-公开
标题翻译： HEPARIN和HEPARAN硫酸盐结合CHIMERIC VECTORS
公开(公告)号：WO2007089632A2
公开(公告)日：2007-08-09
申请号：PCT/US2007/002251
申请日：2007-01-26
申请人： THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL , SAMULSKI, Richard Jude , WU, Zhijian , ASOKAN, Aravind
发明人： SAMULSKI, Richard Jude , WU, Zhijian , ASOKAN, Aravind
IPC分类号： A61K48/00 , C12N15/861 , C12N7/01
CPC分类号： C12N15/86 , A61K48/00 , A61K48/0091 , A61K2039/5256 , C07K14/005 , C12N7/00 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2750/14151 , C12N2810/00
摘要： The present invention is based on the finding that parvovirus (including AAV) capsids can be engineered to incorporate small, selective regions from other parvoviruses that confer desirable properties. In some embodiments, a substitution of a single amino acid that is unique to the AAV6 capsid (Lys-531) among other AAVs that have been characterized to date can confer one or more desirable properties to other AAVs.
摘要翻译：本发明基于以下发现：细小病毒（包括AAV）衣壳可以被工程化以包含来自其他细小病毒的小的选择性区域，其赋予所需的性质。在一些实施方案中，AAV6衣壳（Lys-531）独特的单个氨基酸的替代与目前已被表征的其他AAV之间可以赋予其它AAV所需的一种或多种所需性质。

8. 发明申请

WO2005030261A1 ONCOLYTIC ADENOVIRAL VECTORS ENCODING GM-CSF 审中-公开
标题翻译：编码GM-CSF的ONCOLYTIC ADENOVIRAL VECTORS
公开(公告)号：WO2005030261A1
公开(公告)日：2005-04-07
申请号：PCT/US2003/027380
申请日：2003-08-28
申请人： CELL GENESYS, INC. , ENNIST, David Leonard
发明人： ENNIST, David Leonard
IPC分类号： A61K48/00
CPC分类号： C07K14/535 , A61K38/193 , A61K48/00 , C12N7/00 , C12N15/86 , C12N2710/10343 , C12N2810/00 , C12N2810/405
摘要： Selectively replicating oncolytic adenoviral vectors encoding GM-CSF are provided. The oncolytic adenoviral vectors are useful for expressing GM-CSF in transduced cells and in methods selectively killing a neoplastic cell in a cell population. The vectors are also useful for treating cancer.
摘要翻译：提供选择性复制编码GM-CSF的溶瘤腺病毒载体。溶瘤腺病毒载体可用于在转导的细胞中表达GM-CSF和在细胞群中选择性杀死肿瘤细胞的方法。载体也可用于治疗癌症。

9. 发明申请

WO2015088643A1 METHODS AND COMPOSITIONS FOR THE TARGETED MODIFICATION OF A GENOME 审中-公开
标题翻译：基因组目标修饰的方法和组合
公开(公告)号：WO2015088643A1
公开(公告)日：2015-06-18
申请号：PCT/US2014/060788
申请日：2014-10-15
申请人： REGENERON PHARMACEUTICALS, INC.
发明人： FRENDEWEY, David , AUERBACH, Wojtek , LAI, Ka-Man Venus , MUJICA, Alexander O. , LEE, Jeffrey D. , DROGUETT, Gustavo , TRZASKA, Sean , HUNT, Charleen , GAGLIARDI, Anthony , KUNO, Junko , VALENZUELA, David M. , YANCOPOULOS, George D.
IPC分类号： C12N15/85 , C12N9/22
CPC分类号： C12N15/8509 , A01K67/0276 , A01K67/0278 , A01K2217/072 , A01K2217/075 , A01K2227/105 , A01K2267/0362 , A01K2267/0387 , C12N9/22 , C12N15/1024 , C12N15/85 , C12N15/907 , C12N2015/8527 , C12N2800/40 , C12N2810/00 , C12N2810/10 , C12N2810/40 , C12N2999/007
摘要： Compositions and methods are provided for modifying a genomic locus of interest in a eukaryotic cell, a mammalian cell, a human cell or a non-human mammalian cell using a large targeting vector (LTVEC) comprising various endogenous or exogenous nucleic acid sequences as described herein. Further methods combine the use of the LTVEC with a CRISPR/Cas system. Compositions and methods for generating a genetically modified non-human animal comprising one or more targeted genetic modifications in their germline are also provided.
摘要翻译：提供组合物和方法用于使用包含本文所述的各种内源或外源核酸序列的大靶向载体（LTVEC）修饰真核细胞，哺乳动物细胞，人细胞或非人哺乳动物细胞中的感兴趣的基因组座位。。其他方法将LTVEC与CRISPR / Cas系统的使用相结合。还提供了用于产生遗传修饰的非人动物的组合物和方法，其包含其种系中的一种或多种靶向遗传修饰。

10. 发明申请

WO2009050573A2 FUNCTIONAL ASSAY TO INVESTIGATE UNCLASSIFIED SEQUENCE VARIANTS OF MISMATCH REPAIR GENES 审中-公开
标题翻译：调查错配维修基因未经排序的序列变量的功能测定
公开(公告)号：WO2009050573A2
公开(公告)日：2009-04-23
申请号：PCT/IB2008002758
申请日：2008-10-16
申请人： ACADEMISCH ZIEKENHUIS LEIDEN L , HOEBEN ROBERT CORNELIS , VAN DEN WOLLENBERG DIANA JOHANNA MARIA
发明人： HOEBEN ROBERT CORNELIS , VAN DEN WOLLENBERG DIANA JOHANNA MARIA
IPC分类号： C07K14/00
CPC分类号： C07K14/005 , A61K35/13 , A61K2039/525 , A61K2039/5256 , C07K2319/21 , C12N7/00 , C12N2720/12222 , C12N2720/12252 , C12N2740/15043 , C12N2810/00 , C12N2810/40
摘要： The present invention provides a reverse genetics system for viruses belonging to the Reoviridae (i.e. Reoviruses), various uses thereof, genetically modified Reoviruses, Reovirus selection/production and propagation systems, medicaments and vaccines.
摘要翻译：本发明提供了用于属于呼肠杆菌科（Reovirirus）的病毒的反向遗传学系统，其各种用途，遗传修饰的呼肠病毒，呼肠弧病毒选择/生产和繁殖系统，药物和疫苗。

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