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51. 发明申请

WO2017015102A1 COMPOSITIONS AND METHODS FOR ACHIEVING HIGH LEVELS OF TRANSDUCTION IN HUMAN LIVER CELLS 审中-公开
标题翻译：在人肝细胞中实现高水平转移的组合物和方法
公开(公告)号：WO2017015102A1
公开(公告)日：2017-01-26
申请号：PCT/US2016/042472
申请日：2016-07-15
申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： WILSON, James M. , WANG, Lili , WANG, Qiang
IPC分类号： C12N15/861
CPC分类号： C12N15/86 , C07K2319/00 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2750/14151
摘要： Use of a rAAV3B vector to deliver gene products to human hepatocytes is described. The rAAV3B vectors achieve high levels of transduction even in the presence of pre-existing immunity to AAV8 or AAVrh10. Compositions and treatment regimens are described. Also provided are rAAV engineered to facilitate purification and methods of purifying the AAV.
摘要翻译：描述了使用rAAV3B载体将基因产物递送至人肝细胞。即使存在对AAV8或AAVrh10的预先存在的免疫力，rAAV3B载体也实现高水平的转导。描述了组合物和治疗方案。还提供了旨在促进纯化的rAAV和纯化AAV的方法。

52. 发明申请

WO2016176212A1 ENGINEERED HUMAN ANTI-AAV ANTIBODIES AND USES THEREOF 审中-公开
标题翻译：工程人体抗AAV抗体及其用途
公开(公告)号：WO2016176212A1
公开(公告)日：2016-11-03
申请号：PCT/US2016/029374
申请日：2016-04-26
申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： GILES, April , WILSON, James, M.
IPC分类号： C07K16/00 , C12P21/08
CPC分类号： C07K16/081 , C07B2200/11 , C07K2317/14 , C07K2317/21 , C07K2317/33 , C07K2317/565 , C07K2317/76 , C12N7/00 , C12N2750/14143 , C12N2750/14151 , C12Q1/6876 , G01N33/6854
摘要： A method for generating engineered human anti-AAV antibodies is described. Also described are compositions, panels and arrays containing these antibodies and uses thereof.
摘要翻译：描述了产生工程改造的人抗AAV抗体的方法。还描述了含有这些抗体的组合物，面板和阵列及其用途。

53. 发明申请

WO2016176191A1 DUAL AAV VECTOR SYSTEM FOR CRISPR/CAS9 MEDIATED CORRECTION OF HUMAN DISEASE 审中-公开
标题翻译：用于CRISPR / CAS9的双AAV矢量系统介导的人类疾病修正
公开(公告)号：WO2016176191A1
公开(公告)日：2016-11-03
申请号：PCT/US2016/029330
申请日：2016-04-26
申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： WILSON, James, M. , WANG, Lili , YANG, Yang
IPC分类号： A61K48/00 , C12N9/22 , C12N15/113 , C12N15/86 , C12N15/90
CPC分类号： A61K48/0058 , A61P1/16 , C12N9/22 , C12N15/111 , C12N15/113 , C12N15/86 , C12N15/907 , C12N2310/20 , C12N2750/14143 , C12N2830/008
摘要： A dual vector expression system is provided for delivery of CRISPR/Cas9, sgRNA and donor templates for supplying corrected genes or gene sequences to neonatal subjects. An AAV8 system for delivery to proliferating cells such as hepatocytes is described, as are other dual AAV vector systems for targeting to other cells.
摘要翻译：提供双载体表达系统用于递送CRISPR / Cas9，sgRNA和用于向新生儿受试者提供校正基因或基因序列的供体模板。描述了用于递送到增殖细胞如肝细胞的AAV8系统，以及用于靶向其它细胞的其他双AAV载体系统。

54. 发明申请

WO2016057387A1 COMPOSITIONS AND METHODS FOR ISOLATION OF CIRCULATING TUMOR CELLS (CTC) 审中-公开
标题翻译：用于分离循环肿瘤细胞的组合物和方法（CTC）
公开(公告)号：WO2016057387A1
公开(公告)日：2016-04-14
申请号：PCT/US2015/053982
申请日：2015-10-05
申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： DORSEY, Jay, F. , KAO, Gary, D. , HAHN, Stephen, M.
IPC分类号： C12Q1/70 , C12M1/34
CPC分类号： C12Q1/6897 , C12Q1/6886 , C12Q2600/118 , C12Q2600/156
摘要： A method useful for identifying and isolating live circulating tumor cells is described. The method utilizes an adenoviral vector comprising a replication-competent adenovirus in which the El gene region is expressed under the control of a telomerase- specific promoter and further comprises a second expression cassette containing a marker protein, optionally fused to a detectable cell surface marker to permit detection of circulating tumor cells lacking cell surface markers. The method involves combining ex vivo a test sample from a patient suspected of having circulating tumor cells, an adenoviral probe system, and culture media for the cells. The test sample is incubated with the adenoviral system for a sufficient time to permit expression of the reporter protein. The marker gene expression can thereafter be quantitated and the marker- expressing cells may optionally be collected for further analysis.
摘要翻译：描述了一种用于鉴定和分离活的循环肿瘤细胞的方法。该方法使用包含复制性腺病毒的腺病毒载体，其中E1基因区域在端粒酶特异性启动子的控制下表达，并且还包含含有标记蛋白的第二表达盒，任选地与可检测的细胞表面标记物融合允许检测缺乏细胞表面标志物的循环肿瘤细胞。该方法包括从怀疑患有循环肿瘤细胞的患者，腺病毒探针系统和细胞的培养基组合离体测试样品。将测试样品与腺病毒系统孵育足够的时间以允许报告蛋白的表达。此后可以定量标记基因表达，并且可以任选地收集标志物表达细胞用于进一步分析。

55. 发明申请

WO2016040260A1 EDEMA INVARIANT TRACTOGRAPHY 审中-公开
标题翻译： EDEMA无形TRACTOGRAPHY
公开(公告)号：WO2016040260A1
公开(公告)日：2016-03-17
申请号：PCT/US2015/048844
申请日：2015-09-08
申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： VERMA, Ragini , MACYSZYN, Luke , CARUYER, Emmanuel , LECOEUR, Jeremy , ELLIOT, Mark , BREM, Steven
IPC分类号： G01R33/56
CPC分类号： G01R33/56341 , G01R33/5608
摘要： Edema invariant tractography methods are provided. The methods include acquiring data using a multishell, high angular resolution diffusion imaging sequence; (ii) modeling the data using a multi-compartment model; and (iii) reconstructing the fibers through a probabilistic tracking algorithm using a fiber orientation distribution which has streamlines fitted to the output of said probabilistic tracking algorithm.
摘要翻译：提供了水肿不变导管法。这些方法包括使用多壳高角度分辨率扩散成像序列获取数据; （ii）使用多室模型对数据进行建模; 和（iii）通过概率跟踪算法使用具有拟合到所述概率跟踪算法的输出的流线的光纤取向分布来重建光纤。

56. 发明申请

WO2015138348A1 COMPOSITIONS USEFUL IN TREATMENT OF ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY 审中-公开
标题翻译：用于治疗异丙肾上腺素（OTC）缺乏症的组合物
公开(公告)号：WO2015138348A1
公开(公告)日：2015-09-17
申请号：PCT/US2015/019513
申请日：2015-03-09
申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： WANG, Lili , WILSON, James M.
IPC分类号： C07K14/47 , C12N15/861 , C12N9/10 , C12N9/12 , C12N9/78 , A61K48/00
CPC分类号： C12N9/1018 , A61K48/0058 , C12N15/86 , C12N2710/10343 , C12N2740/15043 , C12N2750/14032 , C12N2750/14033 , C12N2750/14043 , C12N2750/14071 , C12N2750/14143 , C12N2800/22 , C12Y201/03003
摘要： Viral vectors comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis cirrhosis in OTCD patients by administering hOTC.
摘要翻译：提供包含工程化hOTC DNA和RNA序列的病毒载体，其在递送至有需要的受试者时可用于治疗高氨血症，鸟氨酸转氨甲酰酶转氨基甲酰酶缺乏症和与之相关的症状。还提供了通过施用hOTC在OTCD患者中使用hOTC治疗肝纤维化肝硬化的方法。

57. 发明申请

WO2012058102A1 SOLUBLE CALCIUM FERTILIZER FORMULATION 审中-公开
标题翻译：可溶钙肥配方
公开(公告)号：WO2012058102A1
公开(公告)日：2012-05-03
申请号：PCT/US2011/057198
申请日：2011-10-21
申请人： MILLER CHEMICAL & FERTILIZER CORPORATION , SMITH, Andrew, G. , SVEC, Charles, H. , FIERY, Michael, D.
发明人： SMITH, Andrew, G. , SVEC, Charles, H. , FIERY, Michael, D.
IPC分类号： C05B17/00 , C05D1/00 , C05D3/00 , C05G3/00
CPC分类号： C05G3/0064 , C05B17/00 , C05D1/00 , C05D3/00 , C05C9/00 , C05D9/02 , C05F11/10
摘要： A concentrated aqueous fertilizer which is readily water-soluble and a method of applying the solubilized dilute fertilizer to crops to enhance crop production are described. The dry fertilizer contains a contains nitrogen, phosphorus and potassium compounds, as well as a mixture of plant growth stimulators including at least one of each of growth promoters, vitamins, amino acids, carbohydrates/polysaccharides, and adjuvants.
摘要翻译：描述了易于水溶性的浓缩水性肥料和将增溶的稀肥施用于作物以增加作物生产的方法。干肥料含有氮，磷和钾化合物，以及包括生长促进剂，维生素，氨基酸，碳水化合物/多糖和佐剂中的至少一种的植物生长刺激剂的混合物。

58. 发明申请

WO2009105084A2 SIMIAN SUBFAMILY C ADENOVIRUSES SADV-40, -31, AND-34 AND USES THEREOF 审中-公开
标题翻译： SIMIAN亚家族C腺病毒SADV-40，-31和-34及其用途
公开(公告)号：WO2009105084A2
公开(公告)日：2009-08-27
申请号：PCT/US2008/013067
申请日：2008-11-24
申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA , ROY, Soumitra , WILSON, James, M. , VANDENBERGHE, Luc, H.
发明人： ROY, Soumitra , WILSON, James, M. , VANDENBERGHE, Luc, H.
IPC分类号： C12N7/00 , C07K14/075 , C12N15/861
CPC分类号： C12N7/00 , C12N2710/10321 , C12N2710/10322 , C12N2710/10343
摘要： A recombinant vector comprises simian adenovirus SAdV-40, SAdV-31 or SAdV-34 sequences and a heterologous gene under the control of regulatory sequences. A cell line which expresses simian adenovirus SAdV-40, SAdV-31 or SAdV-34 gene(s) is also disclosed. Methods of using the vectors and cell lines are provided.
摘要翻译：重组载体包含猿猴腺病毒SAdV-40，SAdV-31或SAdV-34序列和在调节序列控制下的异源基因。还公开了表达猿猴腺病毒SAdV-40，SAdV-31或SAdV-34基因的细胞系。提供使用载体和细胞系的方法。

59. 发明申请

WO2009089237A1 COMPOSITIONS CONTAINING O-SULFATE AND O-PHOSPHATE CONTAINING ARYL SULFONAMIDE DERIVATIVES USEFUL AS BETA-AMYLOID INHIBITORS 审中-公开
标题翻译：含有硫酸钠和含有磺酰亚胺衍生物的O-磷酸酯的组合物可用作β-淀粉样抑制剂
公开(公告)号：WO2009089237A1
公开(公告)日：2009-07-16
申请号：PCT/US2009/030254
申请日：2009-01-07
申请人： WYETH , LI, David, Zenan , SPRINGER, Dane , ELMARAKBY, Sayed , CHANDRASEKARAN, Appavu , KREFT, Anthony, F.
发明人： LI, David, Zenan , SPRINGER, Dane , ELMARAKBY, Sayed , CHANDRASEKARAN, Appavu , KREFT, Anthony, F.
IPC分类号： A61K31/18 , A61K31/661 , C07C305/04 , C07D333/34 , A61K31/381 , A61P35/00
CPC分类号： C07C305/04 , A61K31/18 , A61K31/381 , A61K31/661 , C07D333/34
摘要： A synthetic compound characterized by having the structure of formula I or II or a pharmaceutically acceptable salt and/or hydrate thereof is provided. Formula I and formula II are defined as follows. wherein R 1 is substituted aryl or substituted heteroaryl; R 2 and R 3 are independently selected from the group consisting of CF 3 , substituted phenyl, C 1 -C 4 alkyl, substituted C 1 -C 4 alkyl, (CF 3 ) n C 1 -C 4 alkyl, (CF 3 ) n (substituted C 1 -C 4 alkyl), provided that when R 2 or R 3 is CF 3 , the other is not an unsubstituted alkyl; R 4 and R 4 ,are independently selected from the group consisting of M, C 1 -C 4 alkyl, phenyl, and benzyl, wherein M is a metal ion is selected from the group consisting of sodium, lithium, calcium, magnesium and potassium, or R 4 and R 4, are taken together to form a cyclic structure. Methods of making such compounds and uses thereof are also provided.
摘要翻译：提供了具有式I或II结构或其药学上可接受的盐和/或水合物的合成化合物。式I和式II定义如下。其中R1是取代的芳基或取代的杂芳基; R 2和R 3独立地选自CF 3，取代的苯基，C 1 -C 4烷基，取代的C 1 -C 4烷基，（CF 3）n C 1 -C 4烷基，（CF 3）n（取代的C 1 -C 4烷基），条件是当R 2 或R3是CF3，另一个不是未取代的烷基; R4和R4独立地选自M，C1-C4烷基，苯基和苄基，其中M是金属离子选自钠，锂，钙，镁和钾，或R4和 R4一起形成环状结构。还提供了制备这些化合物的方法及其用途。

60. 发明申请

WO2009012203A1 PROCESSES AND INTERMEDIATES FOR THE PREPARATION OF HETEROCYCLIC SULFONAMIDE COMPOUNDS 审中-公开
标题翻译：用于制备杂环磺酰胺化合物的方法和中间体
公开(公告)号：WO2009012203A1
公开(公告)日：2009-01-22
申请号：PCT/US2008/069938
申请日：2008-07-14
申请人： WYETH , ALIMARDANOV, Asaf , NIKITENKO, Antonia , FEIGELSON, Gregg , POTOSKI, John
发明人： ALIMARDANOV, Asaf , NIKITENKO, Antonia , FEIGELSON, Gregg , POTOSKI, John
IPC分类号： C07C57/58 , C07C215/28 , C07C311/00 , C07D263/22 , C07D409/00
CPC分类号： C07C303/40 , C07C51/083 , C07C51/36 , C07C57/58 , C07C57/60 , C07C213/00 , C07D263/22 , C07D333/34 , C07D409/12 , C07C311/17 , C07C215/28
摘要： Methods for preparing compound of Formula (I) are described, wherein R 1 -R 3 are defined herein, as are methods for preparing the intermediates formed therein. Formula (I) Also described are methods for enantioselectively preparing a chiral compound of the following structure, wherein R 2 and R 3 are defined herein. Formula (II).
摘要翻译：描述了制备式（I）化合物的方法，其中R 1 -R 3在本文中定义，以及制备其中形成的中间体的方法。式（I）还描述了对映选择性制备下列结构的手性化合物的方法，其中R 2和R 3在本文中定义。式（II）。

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