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    SPLICE SWITCHING OLIGOMERS FOR TNF SUPERFAMILY RECEPTORS AND THEIR USE IN TREATMENT OF DISEASE
    1.
    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
    4.
    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：US20120040917A1

    公开(公告)日：2012-02-16

    申请号：US12960296

    申请日：2010-12-03

    申请人： HENRIK ØRUM ,  PETER L. SAZANI

    发明人： HENRIK ØRUM ,  PETER L. SAZANI

    IPC分类号： A61K38/17 ,  C07H21/04 ,  C07H21/02 ,  A61K31/7088 ,  C12P21/00 ,  A61P29/00 ,  C12N15/12 ,  C12N15/63 ,  C12N1/21 ,  C12N1/19 ,  C12N1/15 ,  C12N5/10 ,  C12N5/071 ,  C07K14/715

    CPC分类号： C07H21/04 ,  A61K38/00 ,  C07H19/00 ,  C07H21/02 ,  C07K14/7151 ,  C12N2799/025 ,  C12N2799/026 ,  Y02A50/463

    摘要： The present invention relates to compositions and methods for preparing splice variants of TNFalpha receptor (TNFR) in vivo or in vitro, and the resulting TNFR protein variants. Such variants may be prepared by controlling the splicing of pre-mRNA molecules and regulating protein expression with splice switching oligonucleotides or splice switching oligomers (SSOs). The preferred SSOs according to the invention target exon 7 or 8 of TNFR1 (TNFRSF1A) or TNFR2 (TNFRSF1A) pre-mRNA, typically resulting in the production of TNFR variants which comprise a deletion in part or the entire exon 7 or 8 respectfully. SSOs targeting exon 7 are found to result in a soluble form of the TNFR, which has therapeutic benefit for treatment of inflammatory diseases. The SSO's are characterized in that they are substantially incapable or incapable of recruiting RNaseH.

    摘要翻译： 本发明涉及用于在体内或体外制备TNFα受体（TNFR）的剪接变体的组合物和方法，以及所得的TNFR蛋白质变体。 可以通过控制前mRNA分子的剪接和用剪接切换寡核苷酸或剪接切换寡聚体（SSO）调节蛋白质表达来制备这些变体。 根据本发明的优选SSO靶向TNFR1（TNFRSF1A）或TNFR2（TNFRSF1A）前mRNA的外显子7或8，通常导致在部分或全部外显子7或8中包含缺失的TNFR变体的产生。 发现针对外显子7的SSOs产生可溶形式的TNFR，其具有治疗炎性疾病的治疗益处。 SSO的特点是它们基本上无能力或不能招募RNaseH。

    Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
    5.
    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 。

    Splice Switching Oligomers for TNF Superfamily Receptors and their Use in Treatment of Disease
    8.
    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超家族受体的接头开关寡聚体及其在治疗疾病中的应用

    公开(公告)号：US20090264353A1

    公开(公告)日：2009-10-22

    申请号：US11875277

    申请日：2007-10-19

    申请人： Henrik Orum ,  Peter L. Sazani

    发明人： Henrik Orum ,  Peter L. Sazani

    IPC分类号： A61K38/17 ,  A61K31/70 ,  A61K31/7052 ,  C07H19/00 ,  C07K14/47 ,  C07H21/02 ,  C12N15/85 ,  C12N5/10 ,  C12P21/02 ,  A61P29/00

    CPC分类号： C07H21/04 ,  A61K38/00 ,  C07H19/00 ,  C07H21/02 ,  C07K14/7151 ,  C12N2799/025 ,  C12N2799/026 ,  Y02A50/463

    摘要： The present invention relates to compositions and methods for preparing splice variants of TNFalpha receptor (TNFR) in vivo or in vitro, and the resulting TNFR protein variants. Such variants may be prepared by controlling the splicing of pre-mRNA molecules and regulating protein expression with splice switching oligonucleotides or splice switching oligomers (SSOs). The preferred SSOs according to the invention target exon 7 or 8 of TNFR1 (TNFRSF1A) or TNFR2 (TNFRSF1A) pre-mRNA, typically resulting in the production of TNFR variants which comprise a deletion in part or the entire exon 7 or 8 respectfully. SSOs targeting exon 7 are found to result in a soluble form of the TNFR, which has therapeutic benefit for treatment of inflammatory diseases. The SSO's are characterized in that they are substantially incapable or incapable of recruiting RNaseH.

    摘要翻译： 本发明涉及用于在体内或体外制备TNFα受体（TNFR）的剪接变体的组合物和方法，以及所得的TNFR蛋白质变体。 可以通过控制前mRNA分子的剪接和用剪接切换寡核苷酸或剪接切换寡聚体（SSO）调节蛋白质表达来制备这些变体。 根据本发明的优选SSO靶向TNFR1（TNFRSF1A）或TNFR2（TNFRSF1A）前mRNA的外显子7或8，通常导致在部分或全部外显子7或8中包含缺失的TNFR变体的产生。 发现针对外显子7的SSOs产生可溶形式的TNFR，其具有治疗炎性疾病的治疗益处。 SSO的特点是它们基本上无能力或不能招募RNaseH。