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公开(公告)号：EP4084796A1
公开(公告)日：2022-11-09
申请号：EP21702131.0
申请日：2021-01-04
发明人： GAO, Dadi , MORINI, Elisabetta , TALKOWSKI, Michael, E. , SLAUGENHAUPT, Susan, A. , LI, Wencheng , EFFENBERGER, Kerstin, Annemarie , TROTTA, Christopher, Robert , YU, Yong , GABBETA, Vijayalakshmi , DAKKA, Amal , NARYSHKIN, Nikolai, A.
IPC分类号： A61K31/519 , C12N15/63 , A61P25/28 , A61P3/06
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公开(公告)号：EP4115882A1
公开(公告)日：2023-01-11
申请号：EP22178466.3
申请日：2016-01-15
申请人： The General Hospital Corporation , The United States of America, as represented by The Secretary, Department of Health and Human Services
IPC分类号： A61K31/4196 , A61P25/28 , C07D251/00 , C07D339/06
摘要： Provided herein are compounds useful for improving mRNA splicing in a cell. Exemplary compounds provided herein are useful for improving mRNA splicing in genes comprising at least one exon ending in the nucleotide sequence CAA. Methods for preparing the compounds and methods of treating diseases of the central nervous system are also provided.
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3.发明公开METHODS FOR ALTERING MRNA SPLICING AND TREATING FAMILIAL DYSAUTONOMIA AND OTHER MECHANISTICALLY RELATED DISORDERS 有权ZUSAMMENSETZUNGEN ZUR BEHANDLUNG VONFAMILIÄRERDYSAUTONOMIE DURCHÄNDERUNGDES SPLEISSENS VON MRNA
公开(公告)号：EP1673439A2
公开(公告)日：2006-06-28
申请号：EP04794054.9
申请日：2004-10-01
IPC分类号： C12N1/00
CPC分类号： A61K31/52 , A61K31/353 , A61K31/355 , C12Q1/6883 , C12Q2600/136 , C12Q2600/156 , C12Q2600/158 , A61K2300/00
摘要： This invention relates to methods for altering the splicing of mRNA in cells. In particular, this invention also relates to methods for increasing the ratio of wild type to misspliced forms of mRNA and corresponding encoded proteins in cells possessing a mutant gene encoding either the i) misspliced mRNA corresponding to the mutant protein or ii) a component in the splicing machinery responsible for processing the misspliced mRNA. In addition, this invention relates to treating individuals having a disorder associated with a misspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, by administering to such an individual a cytokinin such as kinetin.
摘要翻译： 本发明涉及改变细胞中mRNA的剪接的方法。 特别地，本发明还涉及用于增加野生型与错合形式的mRNA和相应编码蛋白的比例的方法，所述细胞具有编码与突变蛋白相对应的i）错过的mRNA的突变基因，或ii） 拼接机构负责处理错误的mRNA。 此外，本发明涉及通过向这样的个体施用诸如激动素的细胞分裂素来治疗与错过的mRNA相关的病症的个体，例如家族性自觉神经病或神经纤维瘤病1。
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4.发明授权COMPOSITIONS FOR TREATING FAMILIAL DYSAUTONOMIA BY ALTERING MRNA SPLICING 有权通过改变MRNA拼接治疗家族性错误修复的组合物
公开(公告)号：EP1673439B1
公开(公告)日：2017-12-06
申请号：EP04794054.9
申请日：2004-10-01
CPC分类号： A61K31/52 , A61K31/353 , A61K31/355 , C12Q1/6883 , C12Q2600/136 , C12Q2600/156 , C12Q2600/158 , A61K2300/00
摘要： This invention relates to methods for altering the splicing of mRNA in cells. In particular, this invention also relates to methods for increasing the ratio of wild type to misspliced forms of mRNA and corresponding encoded proteins in cells possessing a mutant gene encoding either the i) misspliced mRNA corresponding to the mutant protein or ii) a component in the splicing machinery responsible for processing the misspliced mRNA. In addition, this invention relates to treating individuals having a disorder associated with a misspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, by administering to such an individual a cytokinin such as kinetin.
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公开(公告)号：EP3244891A1
公开(公告)日：2017-11-22
申请号：EP16737944.5
申请日：2016-01-15
IPC分类号： A61K31/4196 , A61P25/28 , C07D251/00 , C07D339/06
CPC分类号： C07D473/34 , A61P25/28 , C07D405/12 , C07D417/12 , C07D471/04 , C07D473/16 , C07D487/04 , C07D495/04 , C07D513/04
摘要： Provided herein are compounds useful for improving mRNA splicing in a cell. Exemplary compounds provided herein are useful for improving mRNA splicing in genes comprising at least one exon ending in the nucleotide sequence CAA. Methods for preparing the compounds and methods of treating diseases of the central nervous system are also provided.
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