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公开(公告)号：US20120183602A1
公开(公告)日：2012-07-19
申请号：US13357856
申请日：2012-01-25
申请人： Jianxin CHEN , Steven Ansell , Akin Akinc , Joseph Robert Dorkin , Xiaojun Qin , William Cantley , Muthiah Manoharan , Kallanthottathil G. Rajeev , Jayaprakash K. Narayanannair , Muthusamy Jayaraman
发明人： Jianxin CHEN , Steven Ansell , Akin Akinc , Joseph Robert Dorkin , Xiaojun Qin , William Cantley , Muthiah Manoharan , Kallanthottathil G. Rajeev , Jayaprakash K. Narayanannair , Muthusamy Jayaraman
IPC分类号： A61K9/127 , A61K31/7088 , C12N5/02 , A61K47/44
CPC分类号： A61K9/1272 , A61K9/1278 , A61K31/22 , A61K31/7088 , A61K31/712 , A61K31/713 , A61K38/1709 , A61K38/4846 , A61K47/14 , A61K47/549 , A61K47/551 , A61K47/6911 , A61K48/0033 , C07C29/58 , C07C227/18 , C07C227/40 , C07C229/12 , C07D475/04 , C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , Y02A50/463 , A61K2300/00 , C12N2310/3521 , C12N2310/3533
摘要： The invention features a cationic lipid of formula I, an improved lipid formulation comprising a cationic lipid of formula I and corresponding methods of use. Also disclosed are targeting lipids, and specific lipid formulations comprising such targeting lipids.
摘要翻译： 本发明的特征在于式I的阳离子脂质，包含式I的阳离子脂质和相应的使用方法的改进的脂质制剂。 还公开了靶向脂质和包含这种靶向脂质的特定脂质制剂。
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公开(公告)号：US08034376B2
公开(公告)日：2011-10-11
申请号：US12056230
申请日：2008-03-26
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
IPC分类号： A61K9/127
CPC分类号： C07C219/06 , A61K9/1272 , A61K9/1273 , A61K31/325 , A61K47/34 , C07C209/78 , C07C233/36 , C07C271/10 , C07C323/25
摘要： Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.
摘要翻译： 描述了用于施用基于核酸的疗法，例如缔合复合物如脂质体和脂质复合物的组合物和方法。
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公开(公告)号：US20090286851A1
公开(公告)日：2009-11-19
申请号：US12432192
申请日：2009-04-29
申请人： Akin Akinc , Tomoko Nakayama , Muthiah Manoharan , Markus Stoffel
发明人： Akin Akinc , Tomoko Nakayama , Muthiah Manoharan , Markus Stoffel
IPC分类号： A61K31/7105 , C12N5/06
CPC分类号： C12N15/111 , A61K47/554 , A61K47/6919 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2320/32 , C12N2310/3521 , C12N2310/3533
摘要： This invention relates to new compositions comprising at least one of a single or double stranded oligonucleotide, where said oligonucleotide has been conjugated to a lipophile and to which the conjugated oligonucleotide has been preassembled with lipoproteins. These compositions are effectively in delivering oligonucleotides to mammalian tissue where they effect gene silencing.
摘要翻译： 本发明涉及包含单链或双链寡核苷酸中的至少一种的新组合物，其中所述寡核苷酸已经与亲脂体缀合并且缀合的寡核苷酸已经与脂蛋白预组装。 这些组合物有效地将哺乳动物组织中的寡核苷酸递送到基因沉默中。
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公开(公告)号：US20090023673A1
公开(公告)日：2009-01-22
申请号：US12056230
申请日：2008-03-26
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
IPC分类号： A61K47/14 , A61K31/7088
CPC分类号： C07C219/06 , A61K9/1272 , A61K9/1273 , A61K31/325 , A61K47/34 , C07C209/78 , C07C233/36 , C07C271/10 , C07C323/25
摘要： Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.
摘要翻译： 描述了用于施用基于核酸的疗法，例如缔合复合物如脂质体和脂质复合物的组合物和方法。
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公开(公告)号：US08846631B2
公开(公告)日：2014-09-30
申请号：US13521597
申请日：2011-01-14
申请人： Eric G. Marcusson , Balkrishen Bhat , Peter Linsley , Akin Akinc
发明人： Eric G. Marcusson , Balkrishen Bhat , Peter Linsley , Akin Akinc
CPC分类号： C12N15/113 , A61K31/713 , A61K45/06 , A61K48/00 , C12N2310/141
摘要： Provided herein are compositions comprising oligomeric compounds. In certain embodiments, the oligomeric compounds are useful as miRNA mimics. The oligomeric compounds may mimic the activity of miR-34. Also provided herein are methods for the treatment of cancer.
摘要翻译： 本文提供包含低聚化合物的组合物。 在某些实施方案中，寡聚化合物可用作miRNA模拟物。 寡聚化合物可以模拟miR-34的活性。 本文还提供了治疗癌症的方法。
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26.发明授权公开(公告)号：US08664193B2
公开(公告)日：2014-03-04
申请号：US13619657
申请日：2012-09-14
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/314 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2310/3521 , C12Y304/21021
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Factor VII gene.
摘要翻译： 本发明涉及用于抑制因子VII基因表达的双链核糖核酸（dsRNA）。
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公开(公告)号：US20130123329A1
公开(公告)日：2013-05-16
申请号：US13521597
申请日：2011-01-14
申请人： Eric G. Marcusson , Balkrishen Bhat , Peter Linsley , Akin Akinc
发明人： Eric G. Marcusson , Balkrishen Bhat , Peter Linsley , Akin Akinc
IPC分类号： A61K48/00 , A61K45/06 , C12N15/113
CPC分类号： C12N15/113 , A61K31/713 , A61K45/06 , A61K48/00 , C12N2310/141
摘要： Provided herein are compositions comprising oligomeric compounds. In certain embodiments, the oligomeric compounds are useful as miRNA mimics. The oligomeric compounds may mimic the activity of miR-34. Also provided herein are methods for the treatment of cancer.
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公开(公告)号：US08324366B2
公开(公告)日：2012-12-04
申请号：US12432192
申请日：2009-04-29
申请人： Akin Akinc , Tomoko Nakayama , Muthiah Manoharan , Markus Stoffel
发明人： Akin Akinc , Tomoko Nakayama , Muthiah Manoharan , Markus Stoffel
CPC分类号： C12N15/111 , A61K47/554 , A61K47/6919 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2320/32 , C12N2310/3521 , C12N2310/3533
摘要： This invention relates to new compositions comprising at least one of a single or double stranded oligonucleotide, where said oligonucleotide has been conjugated to a lipophile and to which the conjugated oligonucleotide has been preassembled with lipoproteins. These compositions are effectively in delivering oligonucleotides to mammalian tissue where they effect gene silencing.
摘要翻译： 本发明涉及包含单链或双链寡核苷酸中的至少一种的新组合物，其中所述寡核苷酸已经与亲脂体缀合并且缀合的寡核苷酸已经与脂蛋白预组装。 这些组合物有效地将哺乳动物组织中的寡核苷酸递送到基因沉默中。
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29.发明授权公开(公告)号：US08222222B2
公开(公告)日：2012-07-17
申请号：US12554231
申请日：2009-09-04
申请人： Pamela Tan , Birgit Bramlage , Maria Frank-Kamenetsky , Kevin Fitzgerald , Akin Akinc , Victor E. Kotelianski
发明人： Pamela Tan , Birgit Bramlage , Maria Frank-Kamenetsky , Kevin Fitzgerald , Akin Akinc , Victor E. Kotelianski
CPC分类号： C12N15/1137 , A61K31/713 , C12N2310/111 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the PCSK9 gene (PCSK9 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by PCSK9 gene expression and the expression of the PCSK9 gene using the pharmaceutical composition; and methods for inhibiting expression of a PCSK9 gene in a cell.
摘要翻译： 本发明涉及用于抑制PCSK9基因（PCSK9基因）表达的双链核糖核酸（dsRNA），其包含长度小于30个核苷酸的核苷酸序列的反义链，其长度通常为19-25个核苷酸 并且其与PCSK9基因的至少一部分基本上互补。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 使用该药物组合物治疗由PCSK9基因表达引起的疾病和PCSK9基因表达的方法; 以及抑制细胞中PCSK9基因表达的方法。
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公开(公告)号：US20120046478A1
公开(公告)日：2012-02-23
申请号：US13211094
申请日：2011-08-16
申请人： Muthiah MANOHARAN , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
发明人： Muthiah MANOHARAN , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
IPC分类号： C07C271/16 , C07C235/74 , C07C69/40
CPC分类号： C07C219/06 , A61K9/1272 , A61K9/1273 , A61K31/325 , A61K47/34 , C07C209/78 , C07C233/36 , C07C271/10 , C07C323/25
摘要： Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.
摘要翻译： 描述了用于施用基于核酸的疗法，例如缔合复合物如脂质体和脂质复合物的组合物和方法。
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