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    Oligonucleotide Inhibitors with Chimeric Backbone and 2-Amino-2'-Deoxyadenosine
    13.
    发明申请
    Oligonucleotide Inhibitors with Chimeric Backbone and 2-Amino-2'-Deoxyadenosine 审中-公开
    具有嵌合骨架和2-氨基-2'-脱氧腺苷的寡核苷酸抑制剂

    公开(公告)号：US20140163085A1

    公开(公告)日：2014-06-12

    申请号：US14001480

    申请日：2012-02-23

    申请人： Rosanne Seguin ,  Nicolay Ferrari

    发明人： Rosanne Seguin ,  Nicolay Ferrari

    IPC分类号： C12N15/113

    CPC分类号： C12N15/1137 ,  A61K31/7125 ,  C07H21/00 ,  C12N15/111 ,  C12N15/1136 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/333 ,  C12N2310/343 ,  C12Y301/04017

    摘要： There is provided herein, an oligonucleotide directed against a target gene, wherein the oligonucleotide is capable of hybridizing to at least a portion of a nucleic acid sequence encoding the gene under stringent conditions, and wherein, at least one nucleotide of the oligonucleotide is 2-amino-2′-deoxyadenosine (DAP); and the internucleoside linkages of the oligonucleotide comprises at least three alternating segments, each segment consisting of either at least one phosphorothioate or at least one phosphodiester bond.

    摘要翻译： 本文提供了针对靶基因的寡核苷酸，其中所述寡核苷酸能够在严格条件下与至少一部分编码该基因的核酸序列杂交，并且其中寡核苷酸的至少一个核苷酸为2- 氨基-2'-脱氧腺苷（DAP）; 并且寡核苷酸的核苷间键包含至少三个交替区段，每个区段由至少一个硫代磷酸酯或至少一个磷酸二酯键组成。

    Allele-specific RNA interference
    16.
    发明授权
    Allele-specific RNA interference 有权
    等位基因特异性RNA干扰

    公开(公告)号：US07892793B2

    公开(公告)日：2011-02-22

    申请号：US11241873

    申请日：2005-09-30

    申请人： Zuoshang Xu

    发明人： Zuoshang Xu

    IPC分类号： C07H21/02 ,  C07H21/04 ,  A61K31/70 ,  C12Q1/68

    CPC分类号： C12N15/1137 ,  A61K38/00 ,  A61K48/00 ,  C12N15/111 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/333 ,  C12N2310/336 ,  C12N2310/53 ,  C12N2320/51 ,  C12Y115/01001

    摘要： Human diseases caused by dominant, gain-of-function mutations develop in heterozygotes bearing one mutant and one wild-type copy of a gene. Because the wild-type gene often performs important functions, whereas the mutant gene is toxic, any therapeutic strategy must selectively inhibit the mutant while retaining wild-type gene expression. The present invention includes methods of specifically inhibiting the expression of a mutant allele, while preserving the expression of a co-expressed wild-type allele using RNAi, a therapeutic strategy for treating genetic disorders associated with dominant, gain-of-function gene mutations. The invention also includes small interfering RNAs (siRNAs) and small hairpin RNAs (shRNAs) that selectively suppress mutant, but not wild-type, expression of copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS). The present invention further provides asysmmetric siRNAs and shRNAs with enhanced efficacy and specificity and mediating RNAi.

    摘要翻译： 在具有一个基因的一个突变体和一个野生型拷贝的杂合子中，由显性增益功能突变引起的人类疾病发生。 因为野生型基因通常具有重要的功能，而突变基因是有毒的，任何治疗策略必须选择性地抑制突变体，同时保留野生型基因表达。 本发明包括特异性抑制突变等位基因表达的方法，同时使用RNAi保护共表达野生型等位基因的表达，用于治疗与显性功能性获得功能基因突变相关的遗传疾病的治疗策略。 本发明还包括小干扰RNA（siRNA）和小发夹RNA（shRNA），其选择性地抑制引起遗传性肌萎缩性侧索硬化（ALS）的铜锌超氧化物歧化酶（SOD1）的突变体而不是野生型表达。 本发明还提供了具有增强的功效和特异性并介导RNAi的不对称siRNA和shRNA。

    Antisense agents combining strongly bound base-modified oligonucleotide and artificial nuclease
    17.
    发明授权
    Antisense agents combining strongly bound base-modified oligonucleotide and artificial nuclease 失效
    结合强结合碱基修饰寡核苷酸和人工核酸酶的反义剂

    公开(公告)号：US07786292B2

    公开(公告)日：2010-08-31

    申请号：US11742384

    申请日：2007-04-30

    申请人： Mati Karelson ,  Mart Saarma ,  Mehis Pilv

    发明人： Mati Karelson ,  Mart Saarma ,  Mehis Pilv

    IPC分类号： C07H21/04 ,  C07H21/00 ,  A61K31/70

    CPC分类号： C12N15/111 ,  C12N2310/11 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/3511 ,  C12N2320/51

    摘要： The present invention provides compounds having a chelating moiety and an oligonucleotide sequence wherein the oligonucleotide includes one or more modified nucleobases, such as hydroxynucleobases. The disclosed compounds are suitable for antisense therapy. The chelating moiety can be complexed to an ion of a lanthanide metal. These compounds are efficient translation inhibitors of nucleic acids and have increased binding affinity for target nucleic acids. The invention also includes compositions and methods of using these compositions as antisense therapy.

    摘要翻译： 本发明提供了具有螯合部分和寡核苷酸序列的化合物，其中寡核苷酸包括一个或多个修饰的核碱基，例如羟基核碱基。 所公开的化合物适用于反义治疗。 螯合部分可以与镧系元素金属的离子络合。 这些化合物是核酸的有效翻译抑制剂，并且具有增加的对靶核酸的结合亲和力。 本发明还包括使用这些组合物作为反义治疗的组合物和方法。

    Antisense Agents Combining Strongly Bound Base-Modified Oligonucleotide and Artificial Nuclease
    20.
    发明申请
    Antisense Agents Combining Strongly Bound Base-Modified Oligonucleotide and Artificial Nuclease 失效
    结合强结合碱基修饰寡核苷酸和人工核酸酶的反义剂

    公开(公告)号：US20070259830A1

    公开(公告)日：2007-11-08

    申请号：US11742384

    申请日：2007-04-30

    申请人： Mati Karelson ,  Mart Saarma ,  Mehis Pilv

    发明人： Mati Karelson ,  Mart Saarma ,  Mehis Pilv

    IPC分类号： A61K48/00 ,  C07H21/04 ,  C07F5/00

    CPC分类号： C12N15/111 ,  C12N2310/11 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/3511 ,  C12N2320/51

    摘要： The present invention provides compounds having a chelating moiety and an oligonucleotide sequence wherein the oligonucleotide includes one or more modified nucleobases, such as hydroxynucleobases. The disclosed compounds are suitable for antisense therapy. The chelating moiety can be complexed to an ion of a lanthanide metal. These compounds are efficient translation inhibitors of nucleic acids and have increased binding affinity for target nucleic acids. The invention also includes compositions and methods of using these compositions as antisense therapy.

    摘要翻译： 本发明提供了具有螯合部分和寡核苷酸序列的化合物，其中寡核苷酸包括一个或多个修饰的核碱基，例如羟基核碱基。 所公开的化合物适用于反义治疗。 螯合部分可以与镧系元素金属的离子络合。 这些化合物是核酸的有效翻译抑制剂，并且具有增加的对靶核酸的结合亲和力。 本发明还包括使用这些组合物作为反义治疗的组合物和方法。