-
公开(公告)号：EP2797592A1
公开(公告)日：2014-11-05
申请号：EP12862080.4
申请日：2012-12-28
发明人： WOLL, Matthew G. , CHEN, Guangming , CHOI, Soongyu , DAKKA, Amal , HUANG, Song , KARP, Gary Mitchell , LEE, Chang-Sun , LI, Chunshi , NARASIMHAN, Jana , NARYSHKIN, Nikolai , PAUSHKIN, Sergey , QI, Hongyan , TURPOFF, Anthony A. , WEETALL, Marla L. , WELCH, Ellen , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/353
CPC分类号： C07D487/04 , C07D231/12 , C07D311/16 , C07D311/18 , C07D405/04 , C07D405/12 , C07D405/14 , C07D413/04 , C07D413/14 , C07D417/04 , C07D417/14 , C07D471/04 , C07D491/04 , C07D495/04 , C07D513/04 , C07D519/00
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.
摘要翻译： 本文提供了化合物，其组合物以及用于治疗脊髓性肌萎缩的用途。 在具体的实施方案中，本文提供了可用于调节SMN2的外显子7包含到从SMN2基因转录的mRNA中的形式的化合物。 在另一个具体的实施方案中，本文提供了可用于调节SMN1的外显子7包含到从SMN1基因转录的mRNA中的形式的化合物。 在又一个实施方案中，本文提供了可用于调节SMN1和SMN2的外显子7包含在分别从SMN1和SMN2基因转录的mRNA中的形式的化合物。
-
公开(公告)号：EP1649002A2
公开(公告)日：2006-04-26
申请号：EP04777457.5
申请日：2004-07-02
IPC分类号： C12N1/00
CPC分类号： C12Q1/6897 , C07K14/47 , C12N9/22
摘要： The invention provides human protein complexes with endonuclease activity. In particular, the invention provides human protein complexes with tRNA splicing endonuclease activity and/or 3' end pre-mRNA endonuclease activity. The invention also provides a splice variant of human Sen2, namely human Sen2deltaEx8, and human protein complexes comprising human Sen2deltaEx8. The human Sen2deltaEx8 complexes have pre-tRNA cleavage activity and/or 3' end pre-mRNA endonuclease activity. The invention also provides human protein complexes with pre-ribosomal RNA cleavage activity. The invention also provides antibodies that immunospecifically bind to a complex described herein or a component thereof, and methods of diagnosing, preventing, treating, managing or ameliorating a disorder utilizing such antibodies. The present invention also provides methods utilizing the complexes described herein, inter alia, in screening, diagnosis, and therapy. The invention further provides methods of preparing and purifying the complexes. The present invention further provides methods of identifying a compound that modulates the expression of a component of a complex described herein, the formation of a complex described herein or the activity of a complex described herein, and methods of preventing, treating, managing or ameliorating a disorder, such as a proliferative disorder, or a symptom thereof utilizing a compound identified in accordance with the methods.
-
公开(公告)号：EP2797592B1
公开(公告)日：2019-08-28
申请号：EP12862080.4
申请日：2012-12-28
发明人： WOLL, Matthew G. , CHEN, Guangming , CHOI, Soongyu , DAKKA, Amal , HUANG, Song , KARP, Gary Mitchell , LEE, Chang-Sun , LI, Chunshi , NARASIMHAN, Jana , NARYSHKIN, Nikolai , PAUSHKIN, Sergey , QI, Hongyan , TURPOFF, Anthony A. , WEETALL, Marla L. , WELCH, Ellen , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/353 , C07D413/14 , C07D405/14 , C07D311/16 , C07D311/18 , C07D231/12 , C07D405/04 , C07D405/12 , C07D413/04 , C07D417/04 , C07D471/04 , C07D487/04 , C07D491/04 , C07D495/04 , C07D513/04 , C07D519/00 , A61P25/00 , C07D417/14
-
-
搜索结果
3 条记录 (耗时 0.015 秒)
