公开(公告)号：WO2019104231A1

公开(公告)日：2019-05-31

申请号：PCT/US2018/062332

申请日：2018-11-21

申请人： FARASSATI, Faris

发明人： FARASSATI, Faris

IPC分类号： A61K35/76 ,  A61K35/00 ,  C12N15/86

CPC分类号： A61K35/763 ,  A01K2207/12 ,  A01K2227/105 ,  A01K2267/0331 ,  A61K38/162 ,  A61K48/0058 ,  A61P35/04 ,  C07K16/28 ,  C12N15/1135 ,  C12N15/62 ,  C12N15/8509 ,  C12N2710/16621 ,  C12N2710/16632 ,  C12N2710/16651

摘要： A recombinant lytic virus transcriptionally targeted against malignant cells. A promoter for a viral gene controlling replication is replaced with a promoter for a malignant factor such that the promoter for the malignant factor controls expression of the viral gene controlling replication. Accordingly, the recombinant lytic virus of the present invention only replicates within and kills cells expressing the malignant factor. In an embodiment, the recombinant lytic virus is a recombinant herpes simplex virus, and the viral gene controlling replication is a herpes alpha gene. In an embodiment, the recombinant lytic virus is transcriptionally targeted against cancer stem cells (CSCs). In an embodiment, the recombinant lytic virus is a recombinant herpes simplex virus-1 (HSV-1) with the promoter of CD133 controlling the expression of infected cell protein-4 (ICP4). In another embodiment, the recombinant lytic virus is a recombinant HSV-1 with the promoter of Ezh2 controlling the expression of ICP4.

公开(公告)号：WO2019033083A1

公开(公告)日：2019-02-14

申请号：PCT/US2018/046383

申请日：2018-08-10

申请人： CITY OF HOPE ,  CALIFORNIA INSTITUTE OF TECHNOLOGY

发明人： MARCUCCI, Guido ,  KUI, Ya-Huei ,  HAN, Si-Ping ,  SCHERER, Lisa ,  GODDARD III, William A. ,  ROSSI, John

IPC分类号： C12N15/11 ,  C12N15/113 ,  C12Q1/6886

CPC分类号： C12N15/111 ,  C12N15/1135 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/3519 ,  C12N2320/50 ,  C12Y305/01098

摘要： Disclosed herein are conditional siRNAs activatable by CBFβ-MYH11 oncogenic gene and use thereof for treating conditions such as acute myeloid leukemia (AML). The conditional siRNAs target MCL-1 or HDAC8.

公开(公告)号：WO2019006354A1

公开(公告)日：2019-01-03

申请号：PCT/US2018/040375

申请日：2018-06-29

申请人： NATIONAL JEWISH HEALTH

发明人： JACOBELLI, Jordan ,  THOMPSON, Scott ,  CHUNG, Jeffrey

IPC分类号： A61P35/02 ,  C07H21/00 ,  C07H21/04 ,  C12N15/09 ,  C12Q1/44 ,  C12Q1/68

CPC分类号： G01N33/57496 ,  A61K31/506 ,  A61K31/573 ,  A61K31/713 ,  A61K39/39558 ,  A61K45/06 ,  A61K2039/804 ,  A61P35/02 ,  C07K16/3069 ,  C07K2317/21 ,  C07K2317/76 ,  C12N15/1135 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2320/31 ,  C12Q1/6886 ,  C12Q2600/106 ,  C12Q2600/112 ,  C12Q2600/158 ,  G01N33/57426 ,  G01N2333/4727 ,  A61K2300/00

摘要： The present invention is related to novel methods for detecting and reducing cancer cell central nervous system colonization and methods of treating subjects having cancers that colonize in the central nervous system.

公开(公告)号：WO2017173320A2

公开(公告)日：2017-10-05

申请号：PCT/US2017/025461

申请日：2017-03-31

申请人： AVIDITY BIOSCIENCES LLC

发明人： HUANG, Hanhua ,  BURKE, Rob

IPC分类号： C12N15/113 ,  A61K31/712

CPC分类号： C07K14/47 ,  C07K14/00 ,  C07K14/4748 ,  C12N15/09 ,  C12N15/1135 ,  C12N2310/00 ,  C12N2310/14 ,  C12N2310/315 ,  C12Q1/68 ,  C12Q1/686

摘要： Disclosed herein are molecules and pharmaceutical compositions that mediate RNA interference against MYC. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that mediate RNA interference against MYC.

摘要翻译： 本文公开了介导针对MYC的RNA干扰的分子和药物组合物。 本文还描述了用于治疗疾病或病症的方法，所述疾病或病症包含介导针对MYC的RNA干扰的分子或药物组合物。

公开(公告)号：WO2017004357A1

公开(公告)日：2017-01-05

申请号：PCT/US2016/040361

申请日：2016-06-30

申请人： CITY OF HOPE

发明人： KORTYLEWSKI, Marcin, Tomasz ,  SWIDERSKI, Piotr, Marek ,  MOREIRA, Dayson, Friaca

IPC分类号： A61K47/48 ,  A61K31/713 ,  A61K31/7105 ,  C07H21/04 ,  C07H21/02 ,  C12N15/11 ,  C12N15/113

CPC分类号： A61K47/549 ,  A61K31/713 ,  A61K45/06 ,  A61K48/00 ,  A61P35/00 ,  A61P35/02 ,  A61P37/00 ,  C12N15/113 ,  C12N15/1135 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3519 ,  C12N2320/30 ,  C12N2320/31 ,  C12N2320/35 ,  C12N2320/51 ,  A61K2300/00

摘要： The present disclosure relates to an isolated compound including a phosphorothioated oligodeoxynucleotide (ODN) sequence conjugated to a short-activating RNA (saRNA) or an antisense oligonucleotide sequence (ASO), compositions of such a compound, and method of treatment of cancer and autoimmune diseases (with or without stimulating immune response), method of immune stimulation, method of activating CEBPA, and method of reducing activity of STAT transcription factor, by one of the disclosed compounds or compositions.

摘要翻译： 本公开涉及包含与短活化RNA（saRNA）或反义寡核苷酸序列（ASO）缀合的硫代磷酸化寡脱氧核苷酸（ODN）序列的分离化合物，这种化合物的组合物，以及治疗癌症和自身免疫疾病的方法 （具有或不具有刺激性免疫应答），免疫刺激方法，活化CEBPA的方法和降低STAT转录因子的活性的方法，由所公开的化合物或组合物之一。

公开(公告)号：WO2016201323A1

公开(公告)日：2016-12-15

申请号：PCT/US2016/037018

申请日：2016-06-10

申请人： BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM

发明人： KALLURI, Raghu ,  MELO, Sonia

IPC分类号： C12N15/113 ,  A61K48/00 ,  A61K31/7088

CPC分类号： A61K9/1271 ,  A61K9/0019 ,  A61K9/127 ,  A61K9/5068 ,  A61K45/06 ,  A61P35/00 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/88 ,  C12N2310/14

摘要： The present invention provides lipid-based nanoparticles ( e.g. , liposomes or exosomes) having CD47 on their surface and comprising a therapeutic agent ( e.g. , a therapeutic protein, an antibody, an inhibitory RNA, and/or a small molecule drug). Furthermore, the present invention provides for use of such lipid-based nanoparticles in therapy.

摘要翻译： 本发明提供在其表面上具有CD47的脂质基纳米颗粒（例如脂质体或外来体），其包含治疗剂（例如治疗性蛋白质，抗体，抑制性RNA和/或小分子药物）。 此外，本发明提供了这种基于脂质的纳米颗粒在治疗中的用途。

公开(公告)号：WO2016115558A3

公开(公告)日：2016-09-01

申请号：PCT/US2016013807

申请日：2016-01-18

申请人： DOIRON BRUNO

发明人： DOIRON BRUNO

IPC分类号： A01N63/00 ,  A01K67/00 ,  C07H21/04 ,  C12N15/00 ,  G01N33/00

CPC分类号： A01K67/0275 ,  A01K2217/056 ,  A01K2217/058 ,  A01K2227/105 ,  A01K2267/0331 ,  C07K14/82 ,  C12N15/1135 ,  C12N2320/31 ,  C12N2740/16043 ,  C12N2310/14 ,  C12N2310/531

摘要： Certain embodiments are directed kits comprising components for producing a mammalian cancer model. In certain aspects the components are expression vectors. In certain embodiments one or more expression vector is engineered to express a KrasG12D polypeptide, a p53 transcriptional suppressor, SMAD4 transcriptional suppressor, p16/CDKN2 A transcriptional suppressor. In certain aspects the transcriptional suppressor is a short hairpin RNA (shRNA) or other nucleic acid used for RNA interference.

摘要翻译： 某些实施方案是包含用于产生哺乳动物癌症模型的组分的定向试剂盒。 在某些方面，组分是表达载体。 在某些实施方案中，一种或多种表达载体被工程化以表达KrasG12D多肽，p53转录抑制子，SMAD4转录抑制子，p16 / CDKN2A转录抑制子。 在某些方面，转录抑制因子是用于RNA干扰的短发夹RNA（shRNA）或其他核酸。

公开(公告)号：WO2016041906A3

公开(公告)日：2016-09-01

申请号：PCT/EP2015070971

申请日：2015-09-14

申请人： TECHNISCHE UNIVERSITÄT DRESDEN

发明人： ANDROUTSELLIS-THEOTOKIS ANDREAS ,  NIKOLAKOPOULOU POLYXENI ,  MASJKUR JIMMY ,  POSER STEVEN WALTER

IPC分类号： C12N15/01 ,  A61K31/713 ,  C07K14/47 ,  C07K14/705 ,  C12N15/11 ,  C12N15/113 ,  G01N33/574 ,  G01N33/68

CPC分类号： C07K14/4702 ,  A61K31/713 ,  C12N15/113 ,  C12N15/1135 ,  C12N2310/11 ,  C12N2310/12 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/16 ,  C12N2310/531 ,  G01N33/6893 ,  G01N2800/042 ,  G01N2800/044

摘要： The present invention relates to an inhibitor of Hairy and Enhancer of Split 3 (Hes3) for use in a method of preventing or treating (i) a disease characterised by insulin resistance or (ii) obesity. Further, the present invention also relates to a method of preventing or treating a disease characterised by insulin resistance or obesity, to a pharmaceutical composition comprising Hes3 and to Hes3 and/or an activator thereof for use in a method of preventing or treating a disease characterised by decreased insulin production. In addition, the present invention relates to a method of preventing or treating a disease characterised by decreased insulin production and to the use of Hes3 as a target in a screen for identifying a compound suitable as a lead compound and/or as a medicament for the treatment of diseases characterised by insulin resistance or of obesity or of diseases characterised by decreased insulin production. Moreover, the present invention relates to in-vitro or ex-vivo methods for identifying a compound suitable as a lead compound and/or as a medicament for the treatment and/or prevention of diseases characterised by insulin resistance or of obesity or of diseases characterised by decreased insulin production. Finally, the present invention also relates to an in-vitro method of eliciting or enhancing insulin production.

摘要翻译： 本发明涉及用于预防或治疗（i）以胰岛素抗性为特征的疾病或（ii）肥胖症的方法中的毛发抑制剂和分裂增强剂3（Hes3）。 此外，本发明还涉及预防或治疗以胰岛素抵抗或肥胖为特征的疾病的方法，涉及包含Hes3和Hes3的药物组合物和/或其活化剂，用于预防或治疗特征为 通过减少胰岛素产生。 另外，本发明涉及预防或治疗以胰岛素产生减少为特征的疾病的方法，以及用于鉴定适合作为先导化合物的化合物和/或其药物的筛选中Hes3的用途的方法 治疗以胰岛素抵抗或肥胖为特征的疾病或以胰岛素产生减少为特征的疾病。 此外，本发明涉及用于鉴定适合作为先导化合物的化合物和/或用于治疗和/或预防特征在于胰岛素抵抗或肥胖或表征为特征的疾病的药物的体外或离体方法 通过减少胰岛素产生。 最后，本发明还涉及引发或增强胰岛素产生的体外方法。

公开(公告)号：WO2016106406A2

公开(公告)日：2016-06-30

申请号：PCT/US2015/067561

申请日：2015-12-28

申请人： NITTO DENKO CORPORATION ,  YING, Wenbin

发明人： YING, Wenbin ,  MINOMI, Kenjirou ,  HARBORTH, Jens ,  TAKAHASHI, Hirokazu ,  TERADA, Erika ,  ZHANG, Jun ,  AHMADIAN, Mohammad

CPC分类号： C07F9/6533 ,  A61K31/713 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/322 ,  C12N2310/344 ,  C12N2310/3515 ,  C12N2310/531 ,  C12N2320/30 ,  C12N2320/32 ,  C12N2320/35 ,  C12N2320/53 ,  C12Q1/02 ,  G01N33/582 ,  C12N2310/3533 ,  C12N2310/3531 ,  C12N2310/321 ,  C12N2310/3521

摘要： This invention provides compounds, compositions and methods for modulating the expression of human GST-π using RNA interference. The RNA interference molecules can be used in methods for preventing or treating diseases such as malignant tumor. A nucleic acid molecule can have a) a polynucleotide sense strand and a polynucleotide antisense strand; b) each strand of the molecule being from 15 to 30 nucleotides in length; c) a contiguous region of from 15 to 30 nucleotides of the antisense strand being complementary to a sequence of an mRNA encoding GST-π; and d) at least a portion of the sense strand can be complementary to at least a portion of the antisense strand, and the molecule has a duplex region of from 15 to 30 nucleotides in length

摘要翻译： 本发明提供了使用RNA干扰调节人GST-π表达的化合物，组合物和方法。 RNA干扰分子可用于预防或治疗恶性肿瘤等疾病的方法。 核酸分子可具有a）多核苷酸有义链和多核苷酸反义链; b）分子的每条链长度为15至30个核苷酸; c）反义链的15至30个核苷酸的连续区域与编码GST-π的mRNA的序列互补; 和d）有义链的至少一部分可以与反义链的至少一部分互补，并且该分子具有长度为15至30个核苷酸的双链体区

公开(公告)号：WO2016011167A1

公开(公告)日：2016-01-21

申请号：PCT/US2015/040597

申请日：2015-07-15

申请人： DANA-FARBER CANCER INSTITUTE, INC., ET AL ,  LIVINGSTON, David ,  LIU, Joyce ,  DRAPKIN, Ronny

发明人： LIVINGSTON, David ,  LIU, Joyce ,  DRAPKIN, Ronny

IPC分类号： C07K16/32 ,  A61P35/00

CPC分类号： C07K16/32 ,  A61K39/3955 ,  A61K45/06 ,  A61K2039/55 ,  C07K2317/34 ,  C07K2317/56 ,  C07K2317/73 ,  C07K2317/76 ,  C12N15/111 ,  C12N15/1135 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2320/31

摘要： Methods of treatment of cancer are provided. In particular, methods of treatment of low-grade serous ovarian cancers by inhibiting signaling of an EGFR family member are provided.

摘要翻译： 提供癌症治疗方法。 特别地，提供了通过抑制EGFR家族成员的信号传导治疗低度浆液性卵巢癌的方法。