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    ANTISENSE OLIGONUCLEOTIDE MODULATION OF RAF GENE EXPRESSION
    4.
    发明申请
    ANTISENSE OLIGONUCLEOTIDE MODULATION OF RAF GENE EXPRESSION 审中-公开
    RAF基因表达的抗体寡核苷酸调节

    公开(公告)号：US20140187601A1

    公开(公告)日：2014-07-03

    申请号：US13956213

    申请日：2013-07-31

    申请人： Isis Pharmaceuticals, Inc.

    发明人： Brett P. Monia

    IPC分类号： C12N15/113

    CPC分类号： C12N15/1137 ,  A61K38/00 ,  C07H21/00 ,  C12N15/1135 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3517 ,  C12N2310/3525 ,  C12N2310/3527 ,  C12N2310/3521

    摘要： Oligonucleotides are provided which are targeted to nucleic acids encoding human raf and capable of inhibiting raf expression. The oligonucleotides may have chemical modifications at one or more positions and may be chimeric oligonucleotides. Methods of inhibiting the expression of human raf using oligonucleotides of the invention are also provided. The present invention further comprises methods of inhibiting hyperproliferation of cells and methods of treating or preventing conditions, including hyperproliferative conditions, associated with raf expression.

    摘要翻译： 提供寡核苷酸，其靶向编码人raf的核酸并能够抑制raf表达。 寡核苷酸可以在一个或多个位置具有化学修饰，并且可以是嵌合寡核苷酸。 还提供了使用本发明的寡核苷酸抑制人raf表达的方法。 本发明还包括抑制细胞过度增殖的方法以及治疗或预防与raf表达相关的病症（包括过度增殖病症）的方法。

    COMPOUNDS AND METHODS FOR MODULATING GENE EXPRESSION
    8.
    发明申请
    COMPOUNDS AND METHODS FOR MODULATING GENE EXPRESSION 有权
    化合物和调节基因表达的方法

    公开(公告)号：US20150344879A1

    公开(公告)日：2015-12-03

    申请号：US14698554

    申请日：2015-04-28

    申请人： ISIS PHARMACEUTICALS, INC.

    发明人： Sanjay Bhanot ,  Richard S. Geary ,  Robert McKay ,  Brett P. Monia ,  Punit P. Seth ,  Andrew M. Siwkowski ,  Eric E. Swayze ,  Edward Wancewicz

    IPC分类号： C12N15/113

    CPC分类号： C12N15/113 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/3515 ,  C12Y203/0102 ,  C12N2310/3521

    摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.

    摘要翻译： 本公开描述了短反义化合物，包括长度为化学修饰的高亲和力单体8-16单体的这类化合物。 某些这样的短反义化合物可用于在增加的效力和改善的治疗指数的细胞，组织和动物中还原靶核酸和/或蛋白质。 因此，本文提供了包含用于在体内降低靶RNA的高亲和力核苷酸修饰的短反义化合物。 这样的短反义化合物在比以前描述的反义化合物低的剂量下是有效的，允许降低毒性和治疗成本。 此外，所述短反义化合物具有更大的口服给药潜力。

    OLIGOMERIC COMPOUNDS COMPRISING BICYCLIC NUCLEOSIDES AND USES THEREOF
    9.
    发明申请
    OLIGOMERIC COMPOUNDS COMPRISING BICYCLIC NUCLEOSIDES AND USES THEREOF 有权
    包含双相核苷酸的低分子化合物及其用途

    公开(公告)号：US20150267195A1

    公开(公告)日：2015-09-24

    申请号：US14435109

    申请日：2013-10-11

    申请人： ISIS PHARMACEUTICALS, INC.

    发明人： Punit P. Seth ,  Susan M. Freier ,  Brett P. Monia ,  Eric E. Swayze

    IPC分类号： C12N15/113

    CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/34 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2320/30 ,  C12N2320/51

    摘要： The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. In certain embodiments, the present invention provides compounds comprising oligonucleotides. In certain embodiments, such oligonucleotides comprise a region having a gapmer sugar motif. In certain embodiments, oligonucleotides comprise one or more type of modified sugar moieties and/or naturally occurring sugar moieties arranged along an oligonucleotide or region thereof in a defined pattern or sugar modification motif.

    摘要翻译： 本发明提供低聚化合物。 某些这样的寡聚化合物可用于与互补核酸杂交，包括但不限于细胞中的核酸。 在某些实施方案中，杂交导致细胞中靶核酸的活性或表达的调节。 在某些实施方案中，本发明提供包含寡核苷酸的化合物。 在某些实施方案中，这样的寡核苷酸包含具有间隔糖基序的区域。 在某些实施方案中，寡核苷酸包含沿着寡核苷酸或其区域以限定的图案或糖改性基序排列的一种或多种类型的修饰的糖部分和/或天然存在的糖部分。

    METHODS AND COMPOSITIONS FOR MODULATING ALPHA-1-ANTITRYPSIN EXPRESSION
    10.
    发明申请
    METHODS AND COMPOSITIONS FOR MODULATING ALPHA-1-ANTITRYPSIN EXPRESSION 有权
    调节ALPHA-1抗体表达的方法和组合物

    公开(公告)号：US20150087691A1

    公开(公告)日：2015-03-26

    申请号：US14386246

    申请日：2013-03-19

    申请人： Isis Pharmaceuticals, Inc.

    发明人： Brett P. Monia ,  Michael L. McCaleb ,  Susan M. Freier ,  Shuling Guo

    IPC分类号： C12N15/113

    CPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  C07K14/8125 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346

    摘要： Disclosed herein are methods for decreasing AIAT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of fibrosis. Disclosed herein are methods for decreasing AIAT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of liver disease, such as, AIATD associated liver disease, and pulmonary disease, such as, AIATD associated pulmonary disease in an individual in need thereof. Methods for inhibiting AIAT mRNA and protein expression can also be used as a prophylactic treatment to prevent individuals at risk for developing a liver disease, such as, AIATD associated liver disease and pulmonary disease, such as, AIATD associated pulmonary disease.

    摘要翻译： 本文公开了减少AIAT mRNA和蛋白质表达并治疗，改善，预防，减缓进展或停止纤维化进展的方法。 本文公开了用于降低AIAT mRNA和蛋白质表达以及治疗，改善，预防，减缓进展或停止肝脏疾病如AIATD相关肝病和肺部疾病（例如个体中的AIATD相关肺疾病）的进展的方法 需要它。 用于抑制AIAT mRNA和蛋白质表达的方法也可用作预防性治疗，以预防患有发生肝病，例如AIATD相关肝病和肺部疾病如AIATD相关肺疾病的风险的个体。