公开(公告)号：US6013785A

公开(公告)日：2000-01-11

申请号：US582326

申请日：1996-01-05

申请人： Thomas C. Bruice ,  Robert O. Dempcy ,  Orn Almarsson

发明人： Thomas C. Bruice ,  Robert O. Dempcy ,  Orn Almarsson

IPC分类号： A61K38/00 ,  C07H19/06 ,  C07H19/16 ,  C07H21/00 ,  C12N15/113 ,  C07H21/02

CPC分类号： C12N15/113 ,  C07H19/06 ,  C07H19/16 ,  C07H21/00 ,  C12N15/1132 ,  A61K38/00 ,  C12N2310/15 ,  C12N2310/152 ,  C12N2310/318

摘要： The invention provides a cationic polynucleoside chain having multiple nucleosides, the nucleosides being coupled together by positively charged guanidyl linkages.

摘要翻译： 本发明提供了具有多个核苷的阳离子多核苷酸链，核苷通过带正电荷的胍基键连接在一起。

公开(公告)号：US06004750A

公开(公告)日：1999-12-21

申请号：US905567

申请日：1997-08-04

申请人： Maxim D. Frank-Kamenetskii ,  Alexei G. Veselkov ,  Vadim V. Demidov

发明人： Maxim D. Frank-Kamenetskii ,  Alexei G. Veselkov ,  Vadim V. Demidov

IPC分类号： C12N15/113 ,  C12Q1/68 ,  C07H21/04 ,  C12N9/00 ,  C12P21/00

CPC分类号： C12N15/113 ,  C12Q1/683 ,  C12Q1/6839 ,  C12N2310/152 ,  C12N2310/3181 ,  C12N2310/3183 ,  C12N2310/33 ,  C12N2310/3513

摘要： The invention relates to nucleic acid clamps and methods for using nucleic acid clamps, for example, to inhibit gene expression or cleavage, or to specifically cleave a target nucleic acid. Nucleic acid clamps are molecular devices which can bind nucleic acids with affinity and specificity and have a recognition sequence as small as seven bases. Nucleic acid clamps can be used to modify the effective recognition sequence of restriction endonucleases, reducing the frequency and enhancing the length of the recognition sequence, but without diminishing specificity. The invention also relates to methods for the use of nucleic acid clamps for the treatment of disorders involving abnormal gene expression.

摘要翻译： 本发明涉及核酸钳位和使用核酸钳夹例如抑制基因表达或切割或特异性切割靶核酸的方法。 核酸钳位是可以以亲和力和特异性结合核酸并具有小至七个碱基的识别序列的分子装置。 核酸钳可用于修饰限制性内切核酸酶的有效识别序列，降低识别序列的频率和增加长度，但不会降低特异性。 本发明还涉及使用核酸钳用于治疗涉及异常基因表达的病症的方法。

公开(公告)号：US5877160A

公开(公告)日：1999-03-02

申请号：US483464

申请日：1995-06-07

申请人： Mary Ellen Harper ,  Tod Mitchell Woolf ,  Lyle John Arnold, Jr.

发明人： Mary Ellen Harper ,  Tod Mitchell Woolf ,  Lyle John Arnold, Jr.

IPC分类号： A61K8/60 ,  A61K47/48 ,  A61Q7/00 ,  C07H21/00 ,  C12N15/113 ,  A61K48/00

CPC分类号： A61K31/70 ,  A61K47/48 ,  A61K8/606 ,  A61Q7/00 ,  C07H21/00 ,  C12N15/113 ,  C12N15/1137 ,  C12N15/1138 ,  C12Y103/99005 ,  C12N2310/15 ,  C12N2310/152 ,  C12N2310/3125

摘要： Compositions and methods of treating androgen-associated hair loss, in particular decreasing the progression of male pattern baldness using a nucleoside Oligomer or Oligomers useful in the described methods are provided.

摘要翻译： 提供治疗雄激素相关脱发的组合物和方法，特别是使用核苷降低男性型秃发的进展在所述方法中有用的低聚物或低聚物。

公开(公告)号：US5473060A

公开(公告)日：1995-12-05

申请号：US087387

申请日：1993-07-02

申请人： Sergei M. Gryaznov ,  David H. Lloyd

发明人： Sergei M. Gryaznov ,  David H. Lloyd

IPC分类号： C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  C12Q1/68

CPC分类号： C12N15/113 ,  C07H21/00 ,  C12Q1/6813 ,  C12N2310/152 ,  C12N2310/315 ,  C12N2310/318 ,  C12N2310/3341 ,  C12N2310/351 ,  C12N2310/3515

摘要： Compounds referred to herein as oligonucleotide clamps are provided that stably bind to target polynucleotides in a sequence-specific manner. The oligonucleotide clamps comprise one or more oligonucleotide moieties capable of specifically binding to a target polynucleotide and one or more pairs of binding moieties covalently linked to the oligonucleotide moieties. In accordance with the invention, upon annealing of the oligonucleotide moieties to the target polynucleotide, the binding moieties of a pair are brought into juxtaposition so that they form a stable covalent or non-covalent linkage or complex. The interaction of the binding moieties of the one or more pairs effectively clamps the specifically annealed oligonucleotide moieties to the target polynucleotide.

摘要翻译： 提供本文中称为寡核苷酸钳位的化合物，其以序列特异性方式稳定地结合靶多核苷酸。 寡核苷酸夹具包含能够特异性结合靶多核苷酸的一个或多个寡核苷酸部分和与寡核苷酸部分共价连接的一对或多对结合部分。 根据本发明，当将寡核苷酸部分退火至靶多核苷酸时，将一对的结合部分并列，使其形成稳定的共价或非共价键或复合物。 一个或多个对的结合部分的相互作用有效地将特异性退火的寡核苷酸部分夹持到靶多核苷酸。

公开(公告)号：US20170283830A1

公开(公告)日：2017-10-05

申请号：US15434978

申请日：2017-02-16

申请人： Yale University ,  Carnegie Mellon University

发明人： W. Mark Saltzman ,  Peter Glazer ,  Raman Bahal ,  Nicole Ali McNeer ,  Danith H. Ly ,  Elias Quijano

IPC分类号： C12N15/90 ,  A61K48/00 ,  A61K9/16 ,  C12N15/11

CPC分类号： C12N15/907 ,  A61K9/1647 ,  A61K48/005 ,  C07K14/003 ,  C12N15/102 ,  C12N15/11 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/15 ,  C12N2310/152 ,  C12N2310/318 ,  C12N2310/3181 ,  C12N2310/334 ,  C12N2310/351 ,  C12N2310/3519

摘要： Compositions and methods for enhancing targeted gene editing and methods of use thereof are disclosed. In the most preferred embodiments, gene editing is carried out utilizing a gene editing composition such as triplex-forming oligonucleotides, CRISPR, zinc finger nucleases, TALENS, or others, in combination with a gene modification potentiating agent such as stem cell factor (SCF), a CHK1 or ATR inhibitor, or a combination thereof. A particular preferred gene editing composition is triplex-forming peptide nucleic acids (PNAs) substituted at the γ position for increased DNA binding affinity. Nanoparticle compositions for intracellular delivery of the gene editing composition are also provided and particular advantageous for use with in vivo applications.

公开(公告)号：US09725724B2

公开(公告)日：2017-08-08

申请号：US14278159

申请日：2014-05-15

申请人： Vivonics, Inc.

发明人： Vladimir Leo Gilman

IPC分类号： C07H21/04 ,  C07H19/04 ,  C12N15/115

CPC分类号： C12N15/113 ,  C12N15/115 ,  C12N2310/152 ,  C12N2310/16 ,  C12N2310/311 ,  C12N2310/3517 ,  C12N2320/52

摘要： The invention generally relates to isolated nucleic acid ligands that are neutral under physiological conditions.

公开(公告)号：US08431544B1

公开(公告)日：2013-04-30

申请号：US12869209

申请日：2010-08-26

申请人： Sudhir Agrawal ,  Ekambar Kandimalla ,  Mallikarjuna Putta ,  Tao Lan ,  Lakshmi Bhagat ,  Daqing Wang ,  Dong Yu

发明人： Sudhir Agrawal ,  Ekambar Kandimalla ,  Mallikarjuna Putta ,  Tao Lan ,  Lakshmi Bhagat ,  Daqing Wang ,  Dong Yu

IPC分类号： C12N15/11 ,  C12Q1/68 ,  C12N5/00 ,  C12N5/02 ,  C07H21/04

CPC分类号： C12N15/1138 ,  C12N2310/11 ,  C12N2310/152 ,  C12N2310/321 ,  C12N2310/3519 ,  C12N2310/51

摘要： The inventors have examined the means for providing more efficacious gene expression blocking compounds. The inventors have discovered new structural features that surprisingly improve the efficacy of gene expression blocking molecules. These features include the presence of multiple 3′ ends and a linker at the 5′ ends. Surprisingly, these features improve the efficacy of the gene expression blocking compounds in a manner that decreases the compound's biologic instability. Even more surprisingly, this effect has been found to be applicable to both DNA and RNA oligonucleotide-based compounds and to have application in traditional antisense and RNAi technologies.

摘要翻译： 本发明人已经研究了提供更有效的基因表达阻断化合物的方法。 发明人已经发现了惊人地改善基因表达阻断分子的功效的新结构特征。 这些特征包括多个3'末端的存在和在5'端的接头。 令人惊讶的是，这些特征以降低化合物生物学不稳定性的方式提高了基因表达阻断化合物的功效。 更令人惊奇的是，这种效应已经被发现适用于基于DNA和RNA的寡核苷酸类化合物，并且应用于传统的反义和RNAi技术。

公开(公告)号：US08067574B2

公开(公告)日：2011-11-29

申请号：US12015325

申请日：2008-01-16

申请人： Avraham Hochberg ,  Imad Matouk

发明人： Avraham Hochberg ,  Imad Matouk

IPC分类号： C07H21/04

CPC分类号： C12N15/1135 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/12 ,  C12N2310/14 ,  C12N2310/152

摘要： Isolated oligonucleotides capable of down-regulating H19 mRNA in cancer cells are disclosed, as well as pharmaceutical compositions that include the oligonucleotides and methods of treatment of cancer using them. Methods of treating cancer comprising administering agents capable of downregulating H19 mRNA in combination with an additional anti-cancer treatment are further disclosed.

摘要翻译： 公开了能够下调癌细胞中H19 mRNA的分离的寡核苷酸，以及包括寡核苷酸的药物组合物和使用它们的癌症治疗方法。 进一步公开了治疗癌症的方法，包括与另外的抗癌治疗相结合的能够下调H19mRNA的给药剂。

公开(公告)号：US20090215860A1

公开(公告)日：2009-08-27

申请号：US11570822

申请日：2005-06-17

申请人： Kevin V. Morris ,  David J. Looney ,  Steven E. Jacobsen

发明人： Kevin V. Morris ,  David J. Looney ,  Steven E. Jacobsen

IPC分类号： A61K31/7105 ,  C12Q1/68 ,  C12Q1/70 ,  C12N5/00

CPC分类号： C12N15/111 ,  C12N15/113 ,  C12N15/1131 ,  C12N15/1132 ,  C12N15/1133 ,  C12N2310/111 ,  C12N2310/152 ,  C12N2310/351 ,  C12N2320/10 ,  C12N2320/30 ,  C12N2799/027

摘要： The invention is directed to compositions and methods for RNA-mediated gene regulation, e.g., transcription regulation, e.g., by transcriptional silencing of genes. In one aspect, the invention provides methods using siRNAs directed at a transcription regulator, e.g., a promoter or enhance sequence, of a gene target molecule. In one aspect, this results in in vivo DNA methylation and/or modification of associated chromatin (e.g., histone proteins) accompanied by and/or partial or complete transcription gene silencing in a cell, such as a mammalian cell, e.g., a human cell.

摘要翻译： 本发明涉及用于RNA介导的基因调节的组合物和方法，例如转录调节，例如通过基因的转录沉默。 在一个方面，本发明提供使用针对基因靶分子的转录调节子例如启动子或增强序列的siRNA的方法。 一方面，这导致在细胞如哺乳动物细胞（例如人细胞）中伴随着和/或部分或完全转录基因沉默的相关染色质（例如，组蛋白）的体内DNA甲基化和/或修饰 。

公开(公告)号：US20090143321A1

公开(公告)日：2009-06-04

申请号：US11994810

申请日：2006-07-06

申请人： Avraham Hochberg ,  Imad Matouk

发明人： Avraham Hochberg ,  Imad Matouk

IPC分类号： A61K31/7105 ,  C07H21/02 ,  A61K31/7088

CPC分类号： C12N15/1135 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/12 ,  C12N2310/14 ,  C12N2310/152

摘要： The present invention provides isolated oligonucleotides capable of down-regulating a level of H19 mRNA in cancer cells. Articles of manufacture comprising agents capable of downregulating H19 mRNA in combination with an additional anti-cancer treatment are disclosed as well as methods of treating cancer by administering same.

摘要翻译： 本发明提供能够下调癌细胞中H19 mRNA水平的分离的寡核苷酸。 公开了包含能够与另外的抗癌治疗相结合的能够下调H19mRNA的试剂的制品，以及通过施用它们治疗癌症的方法。