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1. 发明申请

WO2019115748A1 PHARMACEUTICAL CARRIERS CONTAINING miRNAs FOR USE IN THE TREATMENT OF RENAL CANCER 审中-公开
公开(公告)号：WO2019115748A1
公开(公告)日：2019-06-20
申请号：PCT/EP2018/084907
申请日：2018-12-14
申请人： UNICYTE EV AG
发明人： CAMUSSI, Giovanni , BUSSOLATI, Benedetta , LOPATINA, Tatiana
IPC分类号： C12N15/113 , A61K31/7088
CPC分类号： C12N15/113 , A61K31/7105 , C12N2310/141 , C12N2320/32
摘要： The invention relates to a pharmaceutically acceptable carrier that comprises a microRNA selected from the group consisting of miR-15a, miR-181b, miR-320c, miR-874 and any combination thereof, for use in the treatment of renal cancer. The pharmaceutically acceptable carrier is preferably an extracellular vesicle (EV) derived from an adult stem cell selected from the group consisting of a mesenchymal stem cell (MSC), a non-oval human liver progenitor cell (HLSC) and an adipose stem cell (ASC).

2. 发明申请

WO2019006161A1 TREATMENT OF DEVELOPMENTAL SYNDROMES 审中-公开
公开(公告)号：WO2019006161A1
公开(公告)日：2019-01-03
申请号：PCT/US2018/040084
申请日：2018-06-28
申请人： OVID THERAPEUTICS INC.
发明人： DURING, Matthew
IPC分类号： C12N15/63 , C12N15/85 , C12N15/86 , C12N15/861 , A61P3/04 , A61P3/08 , A61P3/10 , A61B5/055
CPC分类号： A61K48/0016 , A61K31/7105 , A61K48/005 , A61K48/0075 , A61M37/0092 , A61P43/00 , C07K14/475 , C12N15/113 , C12N15/1136 , C12N15/62 , C12N2310/141 , C12N2750/14143
摘要： A method of treating a developmental syndrome in a patient in need thereof includes applying ultrasound to a target location in the patient's brain to enhance permeability of the patient's blood brain barrier at the target location and administering to the patient a vector encoding BDNF for delivery of BDNF to the target location, wherein the method provides improvement in at least one symptom of the developmental syndrome. Also provided is a method of treating a developmental syndrome in a patient in need thereof that includes administering to the patient an effective amount of a vector including (i) a constitutive promoter operatively linked to nucleic acid encoding BDNF, and (ii) a regulatory sequence including an AGRP promoter operatively linked to an interference RNA sequence, wherein the regulatory sequence down regulates expression of BDNF in response to BDNF induced physiological changes, and the method provides improvement in at least one symptom of the developmental syndrome.

3. 发明申请

WO2018022982A1 AGE-ASSOCIATED CLONAL HEMATOPOIESIS ACCELERATES CARDIO-METABOLIC DISEASE DEVELOPMENT 审中-公开
标题翻译：年龄相关的克隆性血细胞生成促进心脏代谢疾病的发展
公开(公告)号：WO2018022982A1
公开(公告)日：2018-02-01
申请号：PCT/US2017/044350
申请日：2017-07-28
申请人： TRUSTEES OF BOSTON UNIVERSITY
发明人： WALSH, Kenneth , FUSTER, Jose
IPC分类号： G01N33/50 , G01N33/53 , G01N33/574 , G01N33/577 , C12N9/02 , A61K39/395 , C07K14/705
CPC分类号： C12N5/0647 , A61K39/395 , A61K2035/124 , A61K2039/505 , C07K16/244 , C07K2317/24 , C07K2317/54 , C07K2317/55 , C12N15/113 , C12N2310/141 , C12Q1/68 , C12Q1/6883 , C12Q2600/156
摘要： As demonstrated herein, a preferential and progressive expansion of a subset of hematopoietic cells bearing somatic mutations in TET2 leads to pro-inflammatory IL-1β signaling at multiple levels, including increased IL-1β transcription, increased NLRP3 inflammasome-mediated IL-1β secretion, and increased IL-1-Receptor 1-mediated IL-1β signaling. Accordingly, provided herein are compositions, methods, and assays for modulating TET2 mutation-mediated IL-1β (interleukin-1β) proinflammatory activity, particularly when caused by somatic mutations in TET2.
摘要翻译：如本文所证明的，在TET2中承载体细胞突变的造血细胞亚组的优先和进行性扩增导致多水平的促炎IL-1β信号传导，包括增加的IL-1β转录，增加的NLRP3 炎症介导的IL-1β分泌和增加的IL-1受体1介导的IL-1β信号传导。因此，本文提供了用于调节TET2突变介导的IL-1β（白细胞介素-1β）促炎症活性的组合物，方法和测定法，特别是当由TET2中的体细胞突变引起时。

4. 发明申请

WO2017192959A2 THERAPEUTICALLY MODULATING APOB AND APOAI 审中-公开
标题翻译：治疗APOB和APOAI
公开(公告)号：WO2017192959A2
公开(公告)日：2017-11-09
申请号：PCT/US2017/031240
申请日：2017-05-05
申请人： THE RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK , HUSSAIN, M., Mahmood , ZHOU, Liye
发明人： HUSSAIN, M., Mahmood , ZHOU, Liye
IPC分类号： A61K48/00
CPC分类号： C12N15/113 , A61K31/7088 , A61P3/06 , A61P9/10 , C12N2310/14 , C12N2310/141 , C12N2320/32 , C12N2320/35
摘要： MicroRNAs can be used to decrease expression of apolipoprotein B (apoB), increase expression of apolipoprotein A (apoA), and decrease expression of NCOR1. Use of these microRNAs can simultaneously reduce LDL and increase HDL in circulation and have applications in prevention and treatment of atherosclerosis, hyperlipidemia, and cardiovascular disease as well as other disorders associated with high apoB and/or low apoAI levels.
摘要翻译：微RNA可用于降低载脂蛋白B（apoB）的表达，增加载脂蛋白A（apoA）的表达，并降低NCOR1的表达。这些microRNA的使用可以同时降低LDL并增加循环中的HDL，并且可用于预防和治疗动脉粥样硬化，高脂血症和心血管疾病以及与高apoB和/或低apoAI水平相关的其他病症。

5. 发明申请

WO2017127750A1 MESSENGER RIBONUCLEIC ACIDS FOR THE PRODUCTION OF INTRACELLULAR BINDING POLYPEPTIDES AND METHODS OF USE THEREOF 审中-公开
标题翻译：用于生产胞内结合多肽的信使核糖核酸及其使用方法
公开(公告)号：WO2017127750A1
公开(公告)日：2017-07-27
申请号：PCT/US2017/014441
申请日：2017-01-20
申请人： MODERNATX, INC.
发明人： HUANG, Eric, Yi-chun , FREDERICK, Josh , MCKINNEY, Kristine , HENDERSON, Christina , CHEUNG-ONG, Kahlin , BOLEN, Joseph , KELSEY, Stephen, Michael , MORIN, Michael , GURUMURTHY, Sushma , BENENATO, Kerry , HOGE, Stephen , MCFADYEN, lAIN , PRESNYAK, Vladimir
IPC分类号： C12N15/67 , C07K14/47 , A61K31/7115 , A61P35/00
CPC分类号： C12N15/113 , C07K2319/70 , C12N2310/113 , C12N2310/141
摘要： The invention features isolated mRNAs encoding at least one intracellular binding domain, including mRNAs comprising one or more modified nucleobase and preferably lacking an encoded scaffold polypeptide, and methods of using the same, for example, for inducing apoptosis and/or treating cancer (e.g., liver cancer or colorectal cancer).
摘要翻译：本发明的特征在于编码至少一个细胞内结合结构域的分离的mRNA，其包括包含一个或多个修饰的核碱基并且优选缺少编码的支架多肽的mRNA，以及使用其的方法例如诱导细胞凋亡和/或治疗癌症（例如肝癌或结肠直肠癌）。

6. 发明申请

WO2017102316A1 microRNA miR-142 AS STEM CELL MARKER 审中-公开
标题翻译： microRNA miR-142作为干细胞标记
公开(公告)号：WO2017102316A1
公开(公告)日：2017-06-22
申请号：PCT/EP2016/079161
申请日：2016-11-29
申请人： EUROPEAN MOLECULAR BIOLOGY LABORATORY
发明人： SLADITSCHEK, Hanna , NEVEU, Pierre
IPC分类号： C12Q1/68 , C12N15/11 , C12N5/0735
CPC分类号： C12Q1/6883 , C12N5/0606 , C12N15/113 , C12N2310/113 , C12N2310/141 , C12N2310/20 , C12N2501/65 , C12Q2600/158 , C12Q2600/178
摘要： The present invention relates to a method for detecting a pluripotent stem cell (PSC) in an undifferentiated state, comprising analysing miRNA 142 expression in a PSC, wherein an increase of the expression level of miRNA 142 is indicative for an undifferentiated state, when compared to a differentiating or differentiated PSC. Further provided is a method for maintaining a pluripotent stem cell (PSC) in an undifferentiated state, comprising promoting the expression or overexpression of miRNA 142 in said stem cell and a method for inducing or promoting the differentiation of a pluripotent stem cell (PSC), comprising inhibiting the expression or function of miRNA 142 in said stem cell. The invention also provides a method for identifying a compound that modulates the differentiation of a pluripotent stem cell (PSC), comprising a) contacting an PSC with a potential modulator compound, and b) detecting the expression and/or function of miRNA 142 in said PSC in response to said modulator com¬ pound, wherein a change in the expression and/or function of miRNA 142 is indicative for a compound that modulates the differentiation in said PSC.
摘要翻译：本发明涉及用于检测未分化状态下的多能干细胞（PSC）的方法，包括分析PSC中的miRNA142表达，其中miRNA142的表达水平的提高指示对于未分化状态，与分化或分化PSC相比。还提供了用于将多能干细胞（PSC）维持在未分化状态的方法，包括促进所述干细胞中miRNA 142的表达或过表达，以及诱导或促进多能干细胞（PSC）分化的方法，包括抑制所述干细胞中miRNA 142的表达或功能。本发明还提供了用于鉴定调节多能干细胞（PSC）分化的化合物的方法，包括a）使PSC与潜在的调节剂化合物接触，和b）检测所述miRNA 142在所述的表达和/或功能中的表达和/或功能 PSC响应所述调制器; 其中miRNA 142的表达和/或功能的变化指示调节所述PSC中的分化的化合物。

7. 发明申请

WO2017070302A3 MODULATION OF MICRORNA 184 TO TREAT PATHOLOGICAL LYMPHANGIOGENESIS 审中-公开
标题翻译： MICRORNA 184调节病理性淋巴血管生成
公开(公告)号：WO2017070302A3
公开(公告)日：2017-06-15
申请号：PCT/US2016057838
申请日：2016-10-20
申请人： UNIV CALIFORNIA
发明人： CHEN LU , GRIMALDO SAMMY
IPC分类号： C12N15/113 , C12Q1/68
CPC分类号： A61K31/7088 , C12N15/113 , C12N2310/141
摘要： The present invention provides in certain embodiments a method of suppressing pathological lymphatic formation in a tissue or organ in a mammal in need thereof comprising administering a therapeutic agent comprising Mir-184, pri-Mir-184, a Mir-184 mimic, or a vector comprising an expression cassette comprising a promoter and a nucleic acid encoding Mir-184, pri-Mir-184, or a Mir-184 mimic.
摘要翻译：在某些实施方案中，本发明提供了抑制有需要的哺乳动物的组织或器官中的病理性淋巴形成的方法，其包括施用包含Mir-184，pri-Mir-184，Mir-184模拟物或载体的治疗剂包含含有启动子和编码Mir-184，pri-Mir-184或Mir-184模拟物的核酸的表达盒。

8. 发明申请

WO2016115516A8 METHODS TO PREVENT OR TREAT PERIODONTITIS OR PERI-IMPLANTITIS 审中-公开
标题翻译：预防或治疗牙周炎或周围炎的方法
公开(公告)号：WO2016115516A8
公开(公告)日：2017-06-15
申请号：PCT/US2016013695
申请日：2016-01-15
申请人： UNIV IOWA RES FOUND , HONG LIU , AMENDT BRAD A , SALEM ALIASGER K , ELANGOVAN SATHEESH , ORTIZ GUSTAVO AVILA , SHARP THAD
发明人： HONG LIU , AMENDT BRAD A , SALEM ALIASGER K , ELANGOVAN SATHEESH , ORTIZ GUSTAVO AVILA , SHARP THAD
IPC分类号： C12N15/11 , C12N5/00
CPC分类号： C12N15/113 , C12N2310/141 , C12N2330/10
摘要： Compositions and methods to prevent, inhibit or treat aveolar or periodontal bone loss, enhance bone regeneration, prevent, inhibit or treat peri-implantitis or periodontitis, e.g., periodontitis or peri-implantitis associated with bone loss, or to prevent, inhibit or treat osteoarthritis are provided.
摘要翻译：预防，抑制或治疗牙槽骨或牙周骨损失的组合物和方法，增强骨再生，预防，抑制或治疗种植体周围炎或牙周炎，例如与骨质流失相关的牙周炎或种植体周围炎，或预防，抑制或治疗骨关节炎被提供。

9. 发明申请

WO2017091196A1 REPLICATION FACTOR C-40 AS A PROGNOSTIC MARKER AND TARGET IN BREAST CANCER 审中-公开
标题翻译：复制因子C-40作为乳腺癌预后标记和靶点
公开(公告)号：WO2017091196A1
公开(公告)日：2017-06-01
申请号：PCT/US2015/062118
申请日：2015-11-23
申请人： RAADYSAN BIOTECH, INC.
发明人： GUPTE, Rakhee, S.
IPC分类号： C12N15/11 , C12N15/113 , C12Q1/68 , G01N33/574
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/141 , C12N2310/531 , C12Q1/6886 , C12Q2600/106 , C12Q2600/118 , C12Q2600/158 , G01N33/57415
摘要： The present disclosure relates generally to cancer and particularly to breast cancer including estrogen sensitive, estrogen resistant and triple negative breast cancer (TNBC), and to methods of diagnosis and prognosis thereof and therapeutic intervention involving replication factor C 40 (RFC40). Methods and assays for evaluating breast cancer are provided. The disclosure also relates to inhibition or modulation of RFC40 in treatment or alleviation of cancer, including breast cancer. RFC40 inhibitors, including siRNAs, miRNAs, and shRNAs, which specifically affect cancer cells, particularly breast cancer cells, are provided.
摘要翻译：本发明一般涉及癌症，特别涉及包括对雌激素敏感，抗雌激素和三阴性乳腺癌（TNBC）的乳腺癌，以及涉及其复制因子C的诊断和预后以及治疗性干预的方法 40（RFC40）。提供了用于评估乳腺癌的方法和测定。本公开还涉及在治疗或缓解癌症（包括乳腺癌）中抑制或调节RFC40。提供了RFC40抑制剂，包括特异性影响癌细胞特别是乳腺癌细胞的siRNA，miRNA和shRNA。

10. 发明申请

WO2017079983A1 USES OF MICRORNA MIR-574-5p-BASED COMPOUNDS AS IMMUNOMODULATORS AND COMPOSITIONS THEREOF 审中-公开
标题翻译：基于MICRORNA MIR-574-5p的化合物作为免疫调节剂及其组合物的用途
公开(公告)号：WO2017079983A1
公开(公告)日：2017-05-18
申请号：PCT/CN2015/094617
申请日：2015-11-13
申请人： AMOIGEN BIOSCIENCE (XIAMEN) COMPANY LIMITED
发明人： YANG, James Y. , WANG, Tao , LUO, Yu , CAI, Jianchun , ZHENG, Feng
IPC分类号： A61K48/00 , C12Q1/68 , A61P35/00 , G01N33/68 , G01N33/96 , G01N33/574 , C12N15/113 , C12N15/117
CPC分类号： A61K48/00 , C12N15/113 , C12N15/1138 , C12N2310/14 , C12N2310/141 , C12N2310/17 , C12N2310/315 , C12N2310/3233 , C12Q1/6883 , C12Q2600/178 , G01N33/574 , C12N2310/321 , C12N2310/3521 , C12N2310/3525 , C12N2310/322 , C12N2310/3533
摘要： Provided are the preventive or therapeutic uses of miR-574-5p, miR-574-5p mimics or derivatives, and miR-574-5p inhibitors as immune modulators, which exert actions through modulating TLR7 and/or TLR8 signaling. Also provided are compositions containing miR-574-5p, miR-574-5p mimics or derivatives, or miR-574-5p inhibitors, and administration methods thereof.
摘要翻译：提供miR-574-5p，miR-574-5p模拟物或衍生物和miR-574-5p抑制剂作为免疫调节剂的预防或治疗用途，其通过调节TLR7和/ 或TLR8信号。还提供了含有miR-574-5p，miR-574-5p模拟物或衍生物或miR-574-5p抑制剂的组合物及其给药方法。

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