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11. 发明申请

WO2017066639A1 COMPOUNDS AND COMPOSITIONS INCLUDING PHOSPHOROTHIOATED OLIGODEOXYNUCLEOTIDE, AND METHODS OF USE THEREOF 审中-公开
标题翻译：包括磷酸氢化寡聚脱氧核苷酸的化合物和组合物及其使用方法
公开(公告)号：WO2017066639A1
公开(公告)日：2017-04-20
申请号：PCT/US2016/057143
申请日：2016-10-14
申请人： CITY OF HOPE
发明人： KORTYLEWSKI, Marcin, Tomasz , SWIDERSKI, Piotr, Marek , MARCUCCI, Guido , ZHANG, Bin , KUO, Ya-Huei
IPC分类号： A61K31/7125 , C12N15/117
CPC分类号： A61K31/7125 , C12N15/111 , C12N2310/113 , C12N2310/141 , C12N2310/17 , C12N2310/315 , C12N2310/3519 , C12N2310/321 , C12N2310/3521
摘要： The present disclosure relates to a compound including a nucleic acid sequence conjugated to an anti-microRNA or a microRNA- mimic or a compound including a modified anti-microRNA sequence, compositions of such a compound, and method of treatment of a disease, and method of suppressing microRNA activity by the disclosed compound or composition.
摘要翻译：本公开涉及包含与抗微RNA或微RNA模拟物缀合的核酸序列或包含修饰的抗微RNA序列的化合物的化合物，所述化合物的组合物和方法治疗疾病的方法，以及通过所公开的化合物或组合物抑制微小RNA活性的方法。

12. 发明申请

WO2017035526A1 METHODS AND COMPOSITIONS FOR INHIBITING DETOXIFICATION RESPONSE 审中-公开
标题翻译：抑制脱氧反应的方法和组合物
公开(公告)号：WO2017035526A1
公开(公告)日：2017-03-02
申请号：PCT/US2016/049255
申请日：2016-08-29
申请人： THE GENERAL HOSPITAL CORPORATION
发明人： RUVKUN, Gary , GOVINDAN, J. Amaranath
IPC分类号： C07K16/18 , C12N15/113 , C12N15/63 , A61K35/741
CPC分类号： C12N15/111 , A61K35/74 , C12N2310/11 , C12N2310/14 , C12N2310/141 , C12N2310/16 , C12N2310/3233 , C12N2310/531 , C12N2320/30
摘要： Disclosed herein are compositions and methods for attenuating detoxification response and related symptoms thereof induced by translation defect. The compositions and methods herein are useful for attenuating detoxification response and/or treat related symptoms thereof in subjects comprising translation defect. The composition and methods herein are also useful for improving pharmacokinetics of a pharmaceutical compound.
摘要翻译：本文公开了减轻由翻译缺陷引起的解毒反应及其相关症状的组合物和方法。本文的组合物和方法可用于减弱包含翻译缺陷的受试者的解毒反应和/或治疗其相关症状。本文的组合物和方法也可用于改善药物化合物的药代动力学。

13. 发明申请

WO2017032776A1 HUMAN MICRORNAS FOR TREATMENT OF MALIGNANT TUMOURS 审中-公开
标题翻译：人类微血管治疗恶性肿瘤
公开(公告)号：WO2017032776A1
公开(公告)日：2017-03-02
申请号：PCT/EP2016/069915
申请日：2016-08-23
申请人： UNIVERSITÄTSKLINIKUM JENA
发明人： MROWKA, Ralf , GÖHRING, Axel
IPC分类号： C12N15/113 , C07K14/47 , A61K48/00
CPC分类号： C07K14/4702 , C12N15/1135 , C12N2310/141
摘要： The invention relates to a nucleic acid molecule for use in a method of treatment of cancer. The nucleic acid molecule comprises a sequence selected from SEQ ID NO 001 to SEQ ID NO 038. The nucleic acid molecules provided are not provided for the treatment of laryngeal cancer.
摘要翻译：本发明涉及用于癌症治疗方法的核酸分子。核酸分子包含选自SEQ ID NO：001至SEQ ID NO：038的序列。所提供的核酸分子不提供用于治疗喉癌。

14. 发明申请

WO2016200997A1 NON-INTEGRATING VIRAL DELIVERY SYSTEM AND METHODS OF USE THEREOF 审中-公开
标题翻译：非整合式传播系统及其使用方法
公开(公告)号：WO2016200997A1
公开(公告)日：2016-12-15
申请号：PCT/US2016/036519
申请日：2016-06-08
申请人： AMERICA GENE TECHNOLOGIES INTERNATIONAL, INC.
发明人： PAUZA, Charles, David , LAHUSEN, Tyler
IPC分类号： C12N15/86 , C12N15/867 , C12N15/113 , A61K48/00
CPC分类号： A61K48/0016 , C07K14/47 , C07K16/00 , C12N15/111 , C12N15/113 , C12N15/1131 , C12N15/85 , C12N15/86 , C12N2310/14 , C12N2310/141 , C12N2310/531 , C12N2320/32 , C12N2330/51 , C12N2710/20022 , C12N2710/20043 , C12N2740/16044 , C12N2820/60
摘要： The present invention relates generally to non-integrating viral delivery system and methods of using the same. The viral delivery systems includes a viral carrier, a heterologous viral episomal origin of replication, a sequence encoding at least one initiator protein specific for the heterologous viral episomal origin of replication, and at least one gene, shRNA, siRNA, miRNA, or other gene-silencing RNA of interest. In certain embodiments, the disclosed system can be used for gene therapy.
摘要翻译：本发明一般涉及非整合型病毒递送系统及其使用方法。病毒递送系统包括病毒载体，异源病毒附加型复制起点，编码至少一种对于异源病毒附加型复制起点特异的起始蛋白的序列，以及至少一种基因，shRNA，siRNA，miRNA或其他基因 - 感兴趣的RNA。在某些实施方案中，所公开的系统可用于基因治疗。

15. 发明申请

WO2016190899A1 PRI-MIRNA LIBRARIES AND METHODS FOR MAKING AND USING PRI-MIRNA LIBRARIES 审中-公开
标题翻译： PRI-MIRNA图书馆和制作和使用PRI-MIRNA图书馆的方法
公开(公告)号：WO2016190899A1
公开(公告)日：2016-12-01
申请号：PCT/US2015/062236
申请日：2015-11-24
申请人： BEEM, Alan, M.H.
发明人： BEEM, Alan, M.H.
IPC分类号： C12N15/63 , C12N15/113
CPC分类号： C12N15/1093 , C12N15/111 , C12N2310/141 , C12N2320/12 , C12N2330/31
摘要： Provided are non-native pri-miRNAs, non-native pri-miRNA libraries, vectors comprising and for the expression of one or more non- native pre-miRNAs or for the expression of one or more miRNAs derived from the processing of one or more pre-miRNAs, and cells comprising one or more non-native pri-miRNAs or one or more miRNAs derived from the processing of such non-native pri-miRNAs. Still further provided are methods for regulating a desired cellular phenotype by expressing one or more pri-miRNAs or one or more miRNAs identified through the screening of a pri-miRNA library.
摘要翻译：提供非天然pri-miRNA，非天然pri-miRNA文库，包含和用于表达一种或多种非天然pre-miRNA的载体或用于表达一种或多种来自加工一种或多种 pre-miRNAs和包含一种或多种非天然pri-miRNA或来源于这种非天然pri-miRNA的加工的一种或多种miRNA的细胞。还提供了通过表达一个或多个pri-miRNA或通过筛选pri-miRNA文库鉴定的一种或多种miRNA来调节所需细胞表型的方法。

16. 发明申请

WO2016161345A1 HYPERBRANCHED POLYMERS AND POLYPLEXES AND DNA OR RNA DELIVERY SYSTEMS INCLUDING THE SAME 审中-公开
标题翻译：高分子聚合物和聚合物和包括其中的DNA或RNA递送系统
公开(公告)号：WO2016161345A1
公开(公告)日：2016-10-06
申请号：PCT/US2016/025670
申请日：2016-04-01
申请人： THE REGENTS OF THE UNIVERSITY OF MICHIGAN
发明人： MA, Peter, X. , ZHANG, Xiaojin
IPC分类号： C08G83/00 , A61K47/48 , C08G63/66 , C08G69/44 , C08G73/04
CPC分类号： A61K47/593 , A61K47/50 , A61K47/60 , A61K47/6937 , A61L27/18 , A61L27/26 , A61L27/44 , A61L27/52 , A61L27/54 , A61L27/56 , A61L27/58 , A61L2300/258 , A61L2300/604 , A61L2430/02 , C08F210/02 , C08G63/66 , C08G73/0206 , C08G73/024 , C08G81/00 , C08G83/00 , C08G83/005 , C08G2210/00 , C08G2230/00 , C12N15/113 , C12N2310/14 , C12N2310/141 , C12N2310/351 , C12N2310/51 , C12N2310/52
摘要： A hyperbranched polymer includes a hyperbranched, hydrophobic molecular core, respective low molecular weight polyethyleneimine chains attached to at least three branches of the hyperbranched, hydrophobic molecular core, and respective polyethylene glycol chains attached to at least two other branches of the hyperbranched, hydrophobic molecular core. Examples of the hyperbranched polymer may be used to form hyperbranched polyplexes, and may be included in DNA or RNA delivery systems.
摘要翻译：超支化聚合物包括超支化疏水分子核心，连接至超支化疏水分子核心的至少三个分支的相应的低分子量聚乙烯亚胺链，以及连接至超支化疏水分子核心的至少两个其它分支的相应聚乙二醇链。超支化聚合物的实例可用于形成超支化聚合物，并且可以包括在DNA或RNA递送系统中。

17. 发明申请

WO2016149612A2 ASSAY FOR TELOMERE LENGTH REGULATORS 审中-公开
标题翻译： TELOMERE长度调节器测定
公开(公告)号：WO2016149612A2
公开(公告)日：2016-09-22
申请号：PCT/US2016/023114
申请日：2016-03-18
申请人： THE JOHNS HOPKINS UNIVERSITY
发明人： GREIDER, Carol, W. , LEE, Stella, S.
IPC分类号： C12Q1/68 , C12N15/86
CPC分类号： A61K31/502 , A61K31/4184 , A61K31/55 , A61K31/7105 , A61K31/711 , A61K31/713 , A61K45/06 , A61P7/00 , A61P7/06 , A61P11/00 , A61P17/00 , A61P35/00 , C12N9/1077 , C12N9/12 , C12N15/1137 , C12N15/86 , C12N2310/11 , C12N2310/14 , C12N2310/141 , C12N2310/3231 , C12N2310/531 , C12Q1/6851 , C12Q1/686 , C12Y204/0203 , C12Y207/11022 , A61K2300/00
摘要： The present invention provides an assay that identifies genes required for telomerase-dependent telomere elongation by measuring the de novo telomere addition at a single chromosome.
摘要翻译：本发明提供了通过测量单个染色体上的从头端粒末端添加来鉴定端粒酶依赖性端粒伸长所需基因的测定法。

18. 发明申请

WO2016149580A2 SENSITIZING AGENT FOR CANCER CHEMOTHERAPY AND RADIATION THERAPY AND USES THEREOF 审中-公开
标题翻译：癌症化疗和放射疗法的敏感剂及其用途
公开(公告)号：WO2016149580A2
公开(公告)日：2016-09-22
申请号：PCT/US2016/023065
申请日：2016-03-18
申请人： THE JOHNS HOPKINS UNIVERSITY
发明人： LUPOLD, Shawn, E. , HATANO, Koji , KUMAR, Binod , DEWEESE, Theodore, L.
IPC分类号： A61K48/00 , A61N5/00
CPC分类号： C12N15/111 , C12N2310/141 , C12N2320/31
摘要： The present disclosure provides compositions and methods whereby specific microRNA (miRNA) is utilized as a means to sensitize cancer cells to radiation or chemotherapy.
摘要翻译：本公开提供了组合物和方法，其中使用特异性微小RNA（miRNA）作为使癌细胞对辐射或化学疗法敏感的手段。

19. 发明申请

WO2016090178A3 PROCESSES FOR PRODUCING EXOSOMES IN REDUCED OXYGEN CULTURE CONDITIONS 审中-公开
标题翻译：在减少氧气文化条件下生产废弃物的方法
公开(公告)号：WO2016090178A3
公开(公告)日：2016-08-18
申请号：PCT/US2015063816
申请日：2015-12-03
申请人： CAPRICOR INC
发明人： KREKE MICHELLE , SMITH RACHEL , IBRAHIM AHMED
IPC分类号： C12N5/077 , A61K31/105 , C12N5/02
CPC分类号： A61K35/34 , A61K9/08 , A61K9/19 , A61K47/02 , C12N5/0657 , C12N15/113 , C12N2310/141 , C12N2320/30 , C12N2320/32 , C12N2330/10 , C12N2500/02 , C12N2533/52
摘要： The invention encompasses methods for generating exosomes comprising culturing cells in less than 20% oxygen for at least 2 days and harvesting exosomes from the cells. The invention further encompasses exosome preparations generated from cells cultured in less than 20% oxygen for at least 2 days.
摘要翻译：本发明包括产生外来体的方法，包括在小于20％的氧中培养至少2天的细胞并从细胞中收获外来体。本发明还包括从在少于20％氧气中培养至少2天的细胞产生的外来体制剂。

20. 发明申请

WO2016061232A3 MULTIPLEXED SHRNAS AND USES THEREOF 审中-公开
标题翻译：多拷贝的SHRNAS及其用途
公开(公告)号：WO2016061232A3
公开(公告)日：2016-08-11
申请号：PCT/US2015055537
申请日：2015-10-14
申请人： UNIV TEXAS TECH SYSTEM , WU HAOQUAN , CHOI JANG-GI
发明人： WU HAOQUAN , CHOI JANG-GI
IPC分类号： A61K31/7105 , C12N15/11 , C12N15/113
CPC分类号： C12N15/1132 , A61K48/00 , A61K48/005 , C12N15/1138 , C12N2310/14 , C12N2310/141 , C12N2310/31 , C12N2310/3519 , C12N2310/51 , C12N2310/531 , C12N2330/51
摘要： Nucleic acid molecules such as shRNA clusters and artificial miRNA clusters are disclosed, Also disclosed are methods of use, compositions, cells, viral particles, and kits relating to the nucleic acid molecules disclosed herein. The disclosure provides, at least in part nucleic acid molecules such as shRNA clusters encoding shRNA-like molecules and artificial miRNA clusters encoding modified pri-miRNA-like molecules. The shRNA clusters and artificial miRNA clusters disclosed herein can be used, for example, to produce artificial RNA molecules, e.g., RNAi molecules. Cells, viral particles, compositions (e.g., pharmaceutical compositions), kits, and methods relating to the nucleic acid molecules, e.g., shRNA clusters and artificial miRNA clusters, are also disclosed. The nucleic acid molecules (e.g., shRNA clusters and artificial miRNA clusters), artificial RNA molecules (e.g., RNAi molecules), cells, viral particles, compositions (e.g., pharmaceutical compositions), and kits and methods disclosed herein can be used to treat or prevent a disease, e.g., HIV infection and/or AIDS.
摘要翻译：公开了核酸分子，例如shRNA簇和人工miRNA簇。还公开了与本文公开的核酸分子有关的使用方法，组合物，细胞，病毒颗粒和试剂盒。本公开提供至少部分核酸分子，例如编码shRNA样分子的shRNA簇和编码修饰的pri-miRNA样分子的人工miRNA簇。本文公开的shRNA簇和人工miRNA簇可用于例如人造RNA分子，例如RNAi分子。还公开了细胞，病毒颗粒，组合物（例如药物组合物），试剂盒和与核酸分子有关的方法，例如shRNA簇和人工miRNA簇。本文公开的核酸分子（例如，shRNA簇和人工miRNA簇），人造RNA分子（例如RNAi分子），细胞，病毒颗粒，组合物（例如药物组合物）和试剂盒和方法可用于治疗或预防艾滋病毒/艾滋病等疾病。

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