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    MODIFIED TGF-BETA OLIGONUCLEOTIDE FOR USE IN A METHOD OF PREVENTING AND/OR TREATING AN OPHTHALMIC DISEASE
    51.
    发明申请
    MODIFIED TGF-BETA OLIGONUCLEOTIDE FOR USE IN A METHOD OF PREVENTING AND/OR TREATING AN OPHTHALMIC DISEASE 有权
    用于预防和/或治疗眼病的方法中的改性的TGF-βA寡核苷酸

    公开(公告)号：US20160040167A1

    公开(公告)日：2016-02-11

    申请号：US14779930

    申请日：2014-03-27

    申请人： ISARNA Therapeutics GmbH

    发明人： Frank JASCHINSKI ,  Michel JANICOT ,  Eugen UHLMANN

    IPC分类号： C12N15/113

    CPC分类号： C12N15/1136 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/322 ,  C12N2310/3533

    摘要： The invention refers to an oligonucleotide consisting of 10 to 20 nucleotides of selected regions of the TGF-beta1, TGF-beta2 or TGF-beta3 nucleic acid sequence, which comprises modified nucleotides such as LNA, ENA, polyalkylene oxide-, 2′-fluoro, 2′-O-methoxy and/or 2′-O-methyl modified nucleotides. The invention further relates to pharmaceutical compositions comprising such oligonucleotide, wherein the composition or the oligonucleotide is used in a method for the prevention and/or treatment of glaucoma, posterior capsular opacification, dry eye, Marfan or Loeys-Dietz syndrome, riboblastoma, choroidcarcinoma, macular degeneration, such as age-related macular degeneration, diabetic macular endma, or cataract.

    摘要翻译： 本发明涉及由TGF-β1，TGF-β2或TGF-β3核酸序列的选定区域的10至20个核苷酸组成的寡核苷酸，其包含修饰的核苷酸，例如LNA，ENA，聚环氧烷 - ，2'-氟 2'-O-甲氧基和/或2'-O-甲基修饰的核苷酸。 本发明还涉及包含这种寡核苷酸的药物组合物，其中所述组合物或寡核苷酸用于预防和/或治疗青光眼，后囊膜混浊，干眼症，Marfan或Loeys-Dietz综合征，成纤维细胞瘤，脉络膜癌， 黄斑变性，如年龄相关性黄斑变性，糖尿病性黄斑内皮或白内障。

    NUCLEIC ACID MODULATORS OF GLYCOPROTEIN VI
    54.
    发明申请
    NUCLEIC ACID MODULATORS OF GLYCOPROTEIN VI 审中-公开
    甘氨酸的核酸调节剂六

    公开(公告)号：US20150344533A1

    公开(公告)日：2015-12-03

    申请号：US14540796

    申请日：2014-11-13

    申请人： REGADO BIOSCIENCES, INC.

    发明人： JULIANA M. LAYZER ,  CHRISTOPHER P. RUSCONI ,  DOUGLAS BROOKS ,  STEVEN ZELENKOFSKE

    IPC分类号： C07K14/435

    CPC分类号： C07K14/435 ,  A61K31/7088 ,  A61K31/7105 ,  A61K45/06 ,  A61K47/60 ,  C07K14/70503 ,  C12N15/115 ,  C12N2310/16 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3521 ,  C12N2310/3533

    摘要： The present invention relates, in general, to a pharmacologic system to modulate the biology of platelets based upon a nucleic acid ligand that can interact with and modulate the activity of platelet glycoprotein GPVI to regulate platelet function. These nucleic acid ligands are also actively reversible using a modulator that inhibits the activity of the nucleic acid ligand to neutralize this pharmacologic effect and thereby restore GPVI function, including collagen binding, platelet adhesion, collagen-induced platelet activation, and collagen-induced platelet aggregation. The invention further relates to compositions comprising the nucleic acid ligand, the ligand and a modulator, methods to generate the nucleic acid ligand and its modulator, as well as methods of using these agents and compositions in medical therapeutic and diagnostic procedures.

    摘要翻译： 本发明一般涉及基于可与血小板糖蛋白GPVI相互作用并调节血小板糖蛋白GPVI的活性以调节血小板功能的核酸配体调节血小板生物学的药理学系统。 这些核酸配体也可以使用抑制核酸配体的活性来中和这种药理作用并由此恢复GPVI功能的调节剂，包括胶原结合，血小板粘附，胶原诱导的血小板活化和胶原诱导的血小板聚集 。 本发明还涉及包含核酸配体，配体和调节剂的组合物，产生核酸配体及其调节剂的方法，以及在医学治疗和诊断程序中使用这些试剂和组合物的方法。

    RNA TARGETED TO BETA CATENIN
    55.
    发明申请
    RNA TARGETED TO BETA CATENIN 审中-公开
    RNA定向于角蛋白

    公开(公告)号：US20150291956A1

    公开(公告)日：2015-10-15

    申请号：US14437923

    申请日：2013-02-19

    申请人： PHASERX, INC.

    发明人： Markus Hossbach ,  Jochen Deckert

    IPC分类号： C12N15/113

    CPC分类号： C12N15/113 ,  C12N15/1135 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/343 ,  C12N2310/346 ,  C12N2310/3533 ,  C12N2310/3521

    摘要： A double stranded RNA (dsRNA) molecule targeted to beta catenin includes a duplex region having a sense region and an antisense region at least substantially complementary to the sense region. The sense region and the antisense region each have between 18 and 30 nucleotides. The antisense region includes a nucleotide sequence that is fully complementary to at least 15 contiguous nucleotides of any one of SEQ ID NOs: 1-24.

    摘要翻译： 靶向β-连环蛋白的双链RNA（dsRNA）分子包括具有至少基本上与感觉区域互补的有义区域和反义区域的双链体区域。 有义区域和反义区域各自具有18至30个核苷酸。 反义区域包括与SEQ ID NO：1-24中任一个的至少15个连续核苷酸完全互补的核苷酸序列。

    Pharmaceutical composition comprising anti-miRNA antisense oligonucleotides
    56.
    发明授权
    Pharmaceutical composition comprising anti-miRNA antisense oligonucleotides 有权
    包含抗miRNA反义寡核苷酸的药物组合物

    公开(公告)号：US09133455B2

    公开(公告)日：2015-09-15

    申请号：US14245557

    申请日：2014-04-04

    申请人： Santaris Pharma A/S

    发明人： Joacim Elmén ,  Phil Kearney ,  Sakari Kauppinen

    IPC分类号： C07H21/04 ,  C12N15/113

    CPC分类号： C12N15/113 ,  C12N2310/113 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3515 ,  C12N2310/3521 ,  C12N2310/3533

    摘要： The invention provides pharmaceutical compositions comprising short single stranded oligonucleotides, of length of between 8 and 26 nucleobases which are complementary to human microRNAs selected from the group consisting of miR19b, miR21, miR122a, miR155 and miR375. The short oligonucleotides are particularly effective at alleviating miRNA repression in vivo. It is found that the incorporation of high affinity nucleotide analogs into the oligonucleotides results in highly effective anti-microRNA molecules which appear to function via the formation of almost irreversible duplexes with the miRNA target, rather than RNA cleavage based mechanisms, such as mechanisms associated with RNaseH or RISC.

    摘要翻译： 本发明提供包含长度为8至26个核碱基的短单链寡核苷酸的药物组合物，其与选自miR19b，miR21，miR122a，miR155和miR375的人微小RNA互补。 短的寡核苷酸在减轻体内抑制miRNA方面特别有效。 发现将高亲和力核苷酸类似物并入寡核苷酸导致高效抗微小RNA分子，其似乎通过与miRNA靶标形成几乎不可逆的双链体而不是基于RNA切割的机制，例如与 RNaseH或RISC。