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    Splice switch oligomers for TNF superfamily receptors and their use in treatment of disease
    1.
    发明申请
    Splice switch oligomers for TNF superfamily receptors and their use in treatment of disease 审中-公开
    用于TNF超家族受体的接头切换寡聚体及其在治疗疾病中的应用

    公开(公告)号：US20070105807A1

    公开(公告)日：2007-05-10

    申请号：US11595485

    申请日：2006-11-10

    申请人： Peter Sazani ,  Ryszard Kole ,  Henrik Orum

    发明人： Peter Sazani ,  Ryszard Kole ,  Henrik Orum

    IPC分类号： A61K48/00 ,  C07H21/02

    CPC分类号： C12N15/1138 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/346 ,  C12N2320/33 ,  C12N2310/3521

    摘要： Methods and compositions are disclosed for controlling expression of TNF receptors (TNFR1 and TNFR2) and of other receptors in the TNFR superfamily using compounds that modulate splicing of pre-mRNA encoding these receptors. More specifically these compounds cause the removal of the transmembrane domains of these receptors and produce soluble forms of the receptor which act as an antagonist to reduce TNF-α activity or activity of the relevant ligand. Reducing TNF-α activity provides a method of treating or ameliorating inflammatory diseases or conditions associated with TNF-α activity. Similarly, diseases associated with other ligands can be treated in like manner. In particular, the compounds of the invention are splice-splice switching oligomers (SSOs) which are small molecules that are stable in vivo, hybridize to the RNA in a sequence specific manner and, in conjunction with their target, are not degraded by RNAse H.

    摘要翻译： 公开了使用调节编码这些受体的前mRNA的剪接的化合物来控制TNFR超家族中TNF受体（TNFR1和TNFR2）和其他受体的表达的方法和组合物。 更具体地，这些化合物引起这些受体的跨膜结构域的去除并产生作为减轻TNF-α活性或相关配体的活性的拮抗剂的可溶形式的受体。 降低TNF-α活性提供了治疗或改善与TNF-α活性相关的炎性疾病或病症的方法。 类似地，可以以相似的方式治疗与其它配体相关的疾病。 特别地，本发明的化合物是作为体内稳定的小分子的剪接切割寡聚体（SSO），以序列特异性方式与RNA杂交，并且与其目标结合不被RNA酶H降解 。

    SPLICE SWITCHING OLIGOMERS FOR TNF SUPERFAMILY RECEPTORS AND THEIR USE IN TREATMENT OF DISEASE
    2.
    发明申请
    SPLICE SWITCHING OLIGOMERS FOR TNF SUPERFAMILY RECEPTORS AND THEIR USE IN TREATMENT OF DISEASE 审中-公开
    TNF超家族受体的SPLICE切换寡核苷酸及其在治疗疾病中的应用

    公开(公告)号：US20110237521A1

    公开(公告)日：2011-09-29

    申请号：US12954250

    申请日：2010-11-24

    申请人： Peter L. Sazani ,  Ryszard Kole ,  Henrik Orum

    发明人： Peter L. Sazani ,  Ryszard Kole ,  Henrik Orum

    IPC分类号： A61K38/14 ,  C12N5/071 ,  A61K31/7088 ,  A61K31/712 ,  C07H21/04 ,  C07H21/02 ,  C07K2/00 ,  A61P17/06 ,  A61P19/02 ,  A61P29/00 ,  A61P1/16

    CPC分类号： C12N15/1138 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/346 ,  C12N2320/33 ,  C12N2310/3521

    摘要： Methods and compositions are disclosed for controlling expression of TNF receptors (TNFR1 and TNFR2) and of other receptors in the TNFR superfamily using compounds that modulate splicing of pre-mRNA encoding these receptors. More specifically these compounds cause the removal of the transmembrane domains of these receptors and produce soluble forms of the receptor which act as an antagonist to reduce TNF-α activity or activity of the relevant ligand. Reducing TNF-α activity provides a method of treating or ameliorating inflammatory diseases or conditions associated with TNF-α activity. Similarly, diseases associated with other ligands can be treated in like manner. In particular, the compounds of the invention are splice-splice switching oligomers (SSOs) which are small molecules that are stable in vivo, hybridize to the RNA in a sequence specific manner and, in conjunction with their target, are not degraded by RNAse H.

    摘要翻译： 公开了使用调节编码这些受体的前mRNA的剪接的化合物来控制TNFR超家族中TNF受体（TNFR1和TNFR2）和其他受体的表达的方法和组合物。 更具体地，这些化合物引起这些受体的跨膜结构域的去除，并产生可溶性形式的受体，其作为降低TNF-α活性或相关配体活性的拮抗剂。 降低TNF-α活性提供了治疗或改善与TNF-α活性相关的炎性疾病或病症的方法。 类似地，可以以相似的方式治疗与其它配体相关的疾病。 特别地，本发明的化合物是作为体内稳定的小分子的剪接切割寡聚体（SSO），以序列特异性方式与RNA杂交，并且与其目标结合不被RNA酶H降解 。

    Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
    3.
    发明申请
    Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease 审中-公开
    用于TNF超家族受体的剪接开关寡聚体及其在治疗疾病中的应用

    公开(公告)号：US20140057968A1

    公开(公告)日：2014-02-27

    申请号：US13794497

    申请日：2013-03-11

    申请人： Peter L. Sazani ,  Ryszard Kole ,  Henrik Orum

    发明人： Peter L. Sazani ,  Ryszard Kole ,  Henrik Orum

    IPC分类号： C12N15/113

    CPC分类号： C12N15/1138 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/346 ,  C12N2320/33 ,  C12N2310/3521

    摘要： Methods and compositions are disclosed for controlling expression of TNF receptors (TNFR1 and TNFR2) and of other receptors in the TNFR superfamily using compounds that modulate splicing of pre-mRNA encoding these receptors. More specifically these compounds cause the removal of the transmembrane domains of these receptors and produce soluble forms of the receptor which act as an antagonist to reduce TNF-α activity or activity of the relevant ligand. Reducing TNF-α activity provides a method of treating or ameliorating inflammatory diseases or conditions associated with TNF-α activity. Similarly, diseases associated with other ligands can be treated in like manner. In particular, the compounds of the invention are splice-splice switching oligomers (SSOs) which are small molecules that are stable in vivo, hybridize to the RNA in a sequence specific manner and, in conjunction with their target, are not degraded by RNAse H.

    摘要翻译： 公开了使用调节编码这些受体的前mRNA的剪接的化合物来控制TNFR超家族中TNF受体（TNFR1和TNFR2）和其他受体的表达的方法和组合物。 更具体地，这些化合物引起这些受体的跨膜结构域的去除并产生作为减轻TNF-α活性或相关配体的活性的拮抗剂的可溶形式的受体。 降低TNF-α活性提供了治疗或改善与TNF-α活性相关的炎性疾病或病症的方法。 类似地，可以以相似的方式治疗与其它配体相关的疾病。 特别地，本发明的化合物是作为体内稳定的小分子的剪接切割寡聚体（SSO），以序列特异性方式与RNA杂交，并且与其目标结合不被RNA酶H降解 。

    Soluble HER2 and HER3 splice variant proteins, splice-switching oligonucleotides, and their use in the treatment of disease
    5.
    发明授权
    Soluble HER2 and HER3 splice variant proteins, splice-switching oligonucleotides, and their use in the treatment of disease 有权
    可溶性HER2和HER3剪接变体蛋白，剪接切换寡核苷酸及其在治疗疾病中的应用

    公开(公告)号：US07884194B2

    公开(公告)日：2011-02-08

    申请号：US12157094

    申请日：2008-06-06

    申请人： Ryszard Kole ,  Peter Sazani ,  Jing Wan

    发明人： Ryszard Kole ,  Peter Sazani ,  Jing Wan

    IPC分类号： C07K14/485 ,  C12N15/11

    CPC分类号： C07K14/71 ,  A61K38/00

    摘要： Soluble epidermal growth factor receptors 2 and 3 (HER2 and HER3) splice variant proteins with HER2 and HER3 antagonist activity and anti-proliferative properties, as well as the corresponding nucleic acids, are provided for treatment of proliferative diseases, in particular cancer. Also provided are compositions and methods for inducing expression of these splice variants, including splice switching oligonucleotides that modulate splicing of pre-mRNA that codes for these receptors.

    摘要翻译： 可溶性表皮生长因子受体2和3（HER2和HER3）剪接具有HER2和HER3拮抗剂活性和抗增殖性质的变体蛋白以及相应的核酸被用于治疗增殖性疾病，特别是癌症。 还提供了用于诱导这些剪接变体表达的组合物和方法，包括调节编码这些受体的前mRNA的剪接的剪接切换寡核苷酸。

    Soluble HER2 and HER3 splice variant proteins, splice-switching oligonucleotides, and their use in the treatment of disease
    8.
    发明申请
    Soluble HER2 and HER3 splice variant proteins, splice-switching oligonucleotides, and their use in the treatment of disease 有权
    可溶性HER2和HER3剪接变体蛋白，剪接切换寡核苷酸及其在治疗疾病中的应用

    公开(公告)号：US20090105139A1

    公开(公告)日：2009-04-23

    申请号：US12157094

    申请日：2008-06-06

    申请人： Ryszard Kole ,  Peter Sazani ,  Jing Wan

    发明人： Ryszard Kole ,  Peter Sazani ,  Jing Wan

    IPC分类号： A61K38/16 ,  C07K14/00 ,  A61K31/7088 ,  A61K31/7105 ,  C12N15/11

    CPC分类号： C07K14/71 ,  A61K38/00

    摘要： Soluble epidermal growth factor receptors 2 and 3 (HER2 and HER3) splice variant proteins with HER2 and HER3 antagonist activity and anti-proliferative properties, as well as the corresponding nucleic acids, are provided for treatment of proliferative diseases, in particular cancer. Also provided are compositions and methods for inducing expression of these splice variants, including splice switching oligonucleotides that modulate splicing of pre-mRNA that codes for these receptors.

    摘要翻译： 可溶性表皮生长因子受体2和3（HER2和HER3）剪接具有HER2和HER3拮抗剂活性和抗增殖性质的变体蛋白以及相应的核酸被用于治疗增殖性疾病，特别是癌症。 还提供了用于诱导这些剪接变体表达的组合物和方法，包括调节编码这些受体的前mRNA的剪接的剪接切换寡核苷酸。

    COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY
    10.
    发明申请
    COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY 有权
    用于处理微电子显微镜的化合物​​和方法

    公开(公告)号：US20120058946A1

    公开(公告)日：2012-03-08

    申请号：US13219401

    申请日：2011-08-26

    申请人： Hong M. Moulton ,  Ryszard Kole

    发明人： Hong M. Moulton ,  Ryszard Kole

    IPC分类号： A61K38/08 ,  A61P9/00 ,  A61P21/00 ,  C07K7/02

    CPC分类号： A61K47/48246 ,  A61K31/7088 ,  A61K47/64 ,  C07K2319/33 ,  C12N15/113 ,  C12N15/87 ,  C12N2310/11 ,  C12N2810/40

    摘要： An antisense compound for use in treating myotonic dystrophy DM1 or DM2, a method of enhancing antisense targeting to heart and quadricep muscles, and a method for treating DM1 or DM2 in a mammalian subject are disclosed. The oligonucleotide has 8-30 bases, with at least 8 contiguous bases being complementary to the polyCUG or polyCCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA in DM1 or DM2, respectively. Conjugated to the oligonucleotide is a cell-penetrating peptide having the sequence (RXRR(B/X)R)2XB, where R is arginine; B is β-alanine; and each X is —C(O)—(CH2)n—NH—, where n is 4-6. The antisense compound is effective to selectively block the sequestration of muscleblind-like 1 protein (MBNL1) and/or CUGBP, in heart and quadricep muscle in a myotonic dystrophy animal model.

    摘要翻译： 公开了用于治疗强直性营养不良DM1或DM2的反义化合物，增强对心脏和四头肌的反义靶向的方法，以及哺乳动物受试者中治疗DM1或DM2的方法。 寡核苷酸具有8-30个碱基，其中至少8个连续碱基分别与DM1或DM2中的肌营养不良肌群蛋白激酶（DMPK）mRNA的3'UTR区域中的polyCUG或polyCCUG重复序列互补。 与寡核苷酸共轭是具有序列（RXRR（B / X）R）2XB的细胞穿透肽，其中R是精氨酸; B是丙氨酸; 并且每个X是-C（O） - （CH 2）n -NH-，其中n是4-6。 反义化合物有效地选择性地阻断强直肌营养不良动物模型中心肌和四头肌中肌肉样蛋白样蛋白（MBNL1）和/或CUGBP的螯合。