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51. 发明授权

US09315813B2 Compositions and methods for inhibition of expression of apolipoprotein C-III (APOC3) genes 有权
标题翻译：用于抑制载脂蛋白C-III（APOC3）基因表达的组合物和方法
公开(公告)号：US09315813B2
公开(公告)日：2016-04-19
申请号：US14125567
申请日：2012-06-21
申请人： Brian Bettencourt , Kevin Fitzgerald , Stuart Milstein , Martin Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi , Muthiah Manoharan , Tuyen Nguyen
发明人： Brian Bettencourt , Kevin Fitzgerald , Stuart Milstein , Martin Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi , Muthiah Manoharan , Tuyen Nguyen
IPC分类号： C12N15/11 , C12N15/113
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/344 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) targeting an APOC3 gene, and methods of using the dsRNA to inhibit expression of APOC3.
摘要翻译：本发明涉及靶向APOC3基因的双链核糖核酸（dsRNA），以及使用dsRNA抑制APOC3表达的方法。

52. 发明授权

US09234202B2 Neutralizing RNA aptamers 有权
标题翻译：中和RNA适体
公开(公告)号：US09234202B2
公开(公告)日：2016-01-12
申请号：US14433386
申请日：2013-10-04
申请人： CONSIGLIO NAZIONALE DELLE RICERCHE
发明人： Laura Cerchia , Gerolama Condorelli , Vittorio De Franciscis
IPC分类号： C12N15/115
CPC分类号： C12N15/115 , C12N2310/16 , C12N2310/322 , C12N2310/344 , C12N2310/3533
摘要： Nuclease-resistant RNA aptamers are provided which are capable of neutralizing PDGFRβ and are therefore useful in the diagnosis and/or therapy of PDGFRβ-associated and hyperproliferative-associated diseases, such as cancer and primary tumor metastasis. RNA aptamers provided herein include a modified synthetic RNA sequence wherein at least one pyrimidine residue is modified to 2′-fluoropyrimidine. Pharmaceutical compositions and diagnostic kits comprising RNA aptamers are also provided.
摘要翻译：提供了能够中和PDGFR和bgr的核酸酶抗性的RNA适体; 因此可用于诊断和/或治疗PDGFR和相关和过度增殖相关疾病，如癌症和原发性肿瘤转移。本文提供的RNA适体包括修饰的合成RNA序列，其中至少一个嘧啶残基被修饰成2'-氟嘧啶。还提供了包含RNA适体的药物组合物和诊断试剂盒。

53. 发明授权

US09222091B2 Therapeutic compositions 有权
标题翻译：治疗组合物
公开(公告)号：US09222091B2
公开(公告)日：2015-12-29
申请号：US14282769
申请日：2014-05-20
申请人： Alnylam Pharmaceuticals, Inc.
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , David Bumcrot
IPC分类号： C12N15/113 , A01K67/027 , C07K14/775 , C12N15/11 , C07H21/02
CPC分类号： C12N15/113 , A01K67/0275 , A01K2217/05 , A01K2227/105 , A01K2267/0362 , A61K31/713 , A61K47/48123 , C07H21/02 , C07K14/775 , C12N15/111 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/344 , C12N2310/3515 , C12N2310/533 , C12N2320/32 , C12N2320/51 , C12N2320/53 , C12N2330/30
摘要： This application relates to therapeutic siRNA agents and methods of making and using the agents.
摘要翻译：本申请涉及治疗性siRNA剂及其制备和使用方法。

54. 发明授权

US09222086B2 Compositions and methods for silencing genes expressed in cancer 有权
标题翻译：沉默癌症基因表达的组成和方法
公开(公告)号：US09222086B2
公开(公告)日：2015-12-29
申请号：US13497789
申请日：2010-09-23
申请人： Adam Judge , Yun-Han Lee , Ian MacLachlan , Snorri S. Thorgeirsson
发明人： Adam Judge , Yun-Han Lee , Ian MacLachlan , Snorri S. Thorgeirsson
IPC分类号： A61K31/70 , C07H21/02 , C07H21/04 , C12N15/113 , C12Q1/68 , A61K31/713 , A61K31/7105
CPC分类号： C12N15/113 , A61K31/7105 , A61K31/713 , C12N15/1137 , C12N2310/14 , C12N2310/344 , C12N2320/31 , C12N2320/32
摘要： The present invention provides therapeutic nucleic acids such as interfering RNA (e.g., siRNA) that target the expression of genes associated with tumorigenesis and/or cell transformation, lipid particles (e.g., nucleic acid-lipid particles) comprising one or more (e.g., a cocktail) of the therapeutic nucleic acids, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles, e.g., for the treatment of a cell proliferative disorder such as cancer.
摘要翻译：本发明提供治疗性核酸，例如靶向与肿瘤发生和/或细胞转化相关基因表达的干扰RNA（例如siRNA），包含一种或多种（例如，一种或多种）的脂质颗粒（例如，核酸 - 脂质颗粒）治疗性核酸的混合物），制备脂质颗粒的方法，以及递送和/或施用脂质颗粒的方法，例如用于治疗细胞增殖性疾病如癌症。

55. 发明授权

US09206420B2 Methods and compositions for the specific inhibition of HIF-1α by double-stranded RNA 有权
标题翻译：通过双链RNA特异性抑制HIF-1α的方法和组合物
公开(公告)号：US09206420B2
公开(公告)日：2015-12-08
申请号：US13942014
申请日：2013-07-15
申请人： Dicerna Pharmaceuticals, Inc.
发明人： Bob Dale Brown , Henryk T. Dudek
IPC分类号： C12N15/113 , A61K48/00 , A61K31/713
CPC分类号： C12N15/113 , A61K31/713 , C12N2310/14 , C12N2310/321 , C12N2310/344
摘要： This invention relates to compounds, compositions, and methods useful for reducing HIF-1α target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
摘要翻译：本发明涉及可用于通过使用dsRNA例如Dicer底物siRNA（DsiRNA）试剂降低HIF-1α靶RNA和蛋白质水平的化合物，组合物和方法。

56. 发明申请

US20150344884A1 INHIBITORY OLIGONUCLEOTIDES AND THEIR USE IN THERAPY 审中-公开
标题翻译：抑制性寡核苷酸及其在治疗中的使用
公开(公告)号：US20150344884A1
公开(公告)日：2015-12-03
申请号：US14760791
申请日：2014-01-13
申请人： SAREPTA THERAPEUTICS, INC.
发明人： Eugen UHLMANN , Marion JURK , Thomas LEHMANN
IPC分类号： C12N15/117
CPC分类号： C12N15/117 , C12N2310/17 , C12N2310/315 , C12N2310/33 , C12N2310/334 , C12N2310/3341 , C12N2310/336 , C12N2310/344 , C12N2310/345 , C12N2310/346 , C12N2320/53 , C12N2310/321 , C12N2310/3521
摘要： Inhibitory oligonucleotide having the general formula: X1 C C N1 N2 N3 X2 N4 N5 GGG N6 X3 N7 (I) are disclosed which can be used in pharmaceutical compositions, whereby in formula (I)C is cytidine or a derivative thereof, whereby the cytidine derivative is selected from the group consisting of 5-methylcytidine, a cytidine-like nucleotide having a chemical modification involving the cytosine base, cytidine nucleoside sugar, or both the cytosine base and the cytidine nucleoside sugar, 2′-O-methylcytidine, 5-bromocytidine, 5-hydroxycytidine, ribocytidine and cytosine-β-D-arabinofuranoside,G is guanosine or a derivative thereof, whereby the guanosine derivative is selected from the group consisting of 7-deazaguanosine, a guanosine-like nucleotide having a chemical modification involving the guanine base, the guanosine nucleoside sugar or both the guanine base and the guanosine nucleoside sugar,X1 and X3 is any nucleotide sequence with 0 to 12 bases and each nucleotide is independent of any other, X2 is any nucleotide sequence having 0 to 3 nucleotides,N1, N2 and N3are each independently any nucleotide,N4 and N7 is a pyrimidine or a modified pyrimidine,N5 is a purin or a modified purin,N6 is a modified pyrimidine, A or a modified purin,wherein at least two of the nucleotides N4, N5, N6 or N7 are modified purins or modified pyrimidines.
摘要翻译：公开了具有以下通式的抑制性寡核苷酸：X1 CC N1 N2 N3 X2 N4 N5 GGG N6 X3 N7（I），其可用于药物组合物中，其中式（I）C为胞苷或其衍生物，由此胞苷衍生物选自5-甲基胞苷，具有涉及胞嘧啶碱基，胞苷核苷糖或胞嘧啶碱和胞苷核苷糖，2'-O-甲基胞苷，5-溴胞苷的化学修饰的胞苷样核苷酸，5-羟基胞苷，核糖胞苷和胞嘧啶-D-阿拉伯呋喃糖苷，G是鸟苷或其衍生物，其中鸟苷衍生物选自7-脱氮鸟苷，具有化学修饰的鸟苷样核苷酸，涉及鸟嘌呤碱基，鸟苷核苷或鸟嘌呤核苷和鸟苷核糖，X1和X3均为0至12个碱基的核苷酸序列，每个核苷酸为ind 任何其他的核苷酸序列，X2是具有0至3个核苷酸的任何核苷酸序列，N1，N2和N3各自独立地为任何核苷酸，N4和N7为嘧啶或修饰的嘧啶，N5为嘌呤或经修饰的嘌呤，N6为经修饰的嘧啶，A或修饰的嘌呤，其中核苷酸N4，N5，N6或N7中的至少两个是修饰的嘌呤或修饰的嘧啶。

57. 发明授权

US09193973B2 Compositions and methods for increasing erythropoietin (EPO) production 有权
标题翻译：增加红细胞生成素（EPO）生产的组合物和方法
公开(公告)号：US09193973B2
公开(公告)日：2015-11-24
申请号：US13992334
申请日：2011-12-09
申请人： William G. Kaelin, Jr. , Victor Kotelianski , William Querbes , Brian Bettencourt
发明人： William G. Kaelin, Jr. , Victor Kotelianski , William Querbes , Brian Bettencourt
IPC分类号： C12N15/11 , C12Q1/68 , C12N15/113
CPC分类号： C12N15/1137 , A61K31/713 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/335 , C12N2310/344 , C12N2320/31 , C12Y114/11002 , C12N2310/3521
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting one or more EGLN genes, EGLN1, EGLN2 and/or EGLN3 and methods of using such dsRNA compositions to inhibit expression of these genes.
摘要翻译：本发明涉及靶向一种或多种EGLN基因，EGLN1，EGLN2和/或EGLN3的双链核糖核酸（dsRNA）组合物以及使用这种dsRNA组合物抑制这些基因表达的方法。

58. 发明申请

US20150284719A1 METHODS AND COMPOSITIONS INVOLVING MIRNA AND MIRNA INHIBITOR MOLECULES 审中-公开
公开(公告)号：US20150284719A1
公开(公告)日：2015-10-08
申请号：US14738706
申请日：2015-06-12
申请人： Asuragen, Inc.
发明人： David BROWN , Lance FORD , Angie CHENG , Rich JARVIS , Mike BYROM , Dmitriy OVCHARENKO , Eric DEVROE , Kevin KELNAR
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K9/127 , A61K31/7088 , A61K31/7105 , A61K31/713 , A61K45/06 , A61N5/10 , C12N15/111 , C12N15/1136 , C12N2310/14 , C12N2310/141 , C12N2310/312 , C12N2310/321 , C12N2310/322 , C12N2310/33 , C12N2310/344 , C12N2310/35 , C12N2310/351 , C12N2310/3527 , C12N2310/3535 , C12N2310/533 , C12N2320/12 , C12N2320/30 , C12N2320/50 , C12N2330/10 , C12Q1/68 , C12Q1/6876 , C12Q2600/158 , C12Q2600/178 , C12N2310/3521
摘要： The present invention concerns methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules. Moreover, the present invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library screening assays.

59. 发明申请

US20150252363A1 HUMAN TOLL-LIKE RECEPTOR INHIBITORS AND METHODS OF USE THEREOF 有权
标题翻译：人类样受体抑制剂及其使用方法
公开(公告)号：US20150252363A1
公开(公告)日：2015-09-10
申请号：US14430876
申请日：2013-09-27
申请人： DYNAVAX TECHNOLOGIES CORPORATION
发明人： Cristiana Guiducci , Karen L. Fearon , Franck Barrat
IPC分类号： C12N15/11
CPC分类号： C12N15/117 , C12N2310/17 , C12N2310/331 , C12N2310/344
摘要： Provided herein are human Toll-like receptor (TLR)-inhibitors and methods for use in individuals having an autoimmune disease or an inflammatory disorder. The TLR inhibitors of the present disclosure are polynucleotides comprising an inhibitory motif for one or more of TLR7, TLR8 and TLR9.
摘要翻译：本文提供了人Toll样受体（TLR） - 抑制剂和用于具有自身免疫性疾病或炎症性疾病的个体的方法。本公开的TLR抑制剂是包含TLR7，TLR8和TLR9中的一种或多种的抑制性基序的多核苷酸。

60. 发明授权

US09127278B2 Modulation of hepatitis B virus (HBV) expression 有权
公开(公告)号：US09127278B2
公开(公告)日：2015-09-08
申请号：US14464449
申请日：2014-08-20
申请人： Isis Pharmaceuticals, Inc.
发明人： Susan M. Freier
IPC分类号： C12N15/113
CPC分类号： C12N15/1131 , C12N2310/11 , C12N2310/313 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/344 , C12N2310/346 , C12N2320/30 , C12N2310/3525
摘要： Disclosed herein are antisense compounds and methods for decreasing HBV mRNA, DNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate HBV-related diseases, disorders or conditions.

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