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1. 发明申请

US20080090774A1 Nucleoside compounds and their use for treating cancer and diseases associated with somatic mutations 审中-公开
标题翻译：核苷化合物及其用于治疗与体细胞突变相关的癌症和疾病的用途
公开(公告)号：US20080090774A1
公开(公告)日：2008-04-17
申请号：US11906665
申请日：2007-10-02
申请人： Richard Wilde , Paul Kennedy , Neil Almstead , Ellen Welch , James Takasugi , Westley Friesen
发明人： Richard Wilde , Paul Kennedy , Neil Almstead , Ellen Welch , James Takasugi , Westley Friesen
IPC分类号： A61K31/706 , A61P35/00
CPC分类号： C07H19/00
摘要： The invention encompasses nucleoside compounds, compositions comprising the compounds and methods for treating or preventing diseases associated with nonsense mutations of mRNA by administering these compounds or compositions.
摘要翻译：本发明包括核苷化合物，包含该化合物的组合物和通过施用这些化合物或组合物来治疗或预防与无义突变mRNA相关的疾病的方法。

2. 发明申请

US20070203123A1 Use of acetylamino benzoic acid compounds for nonsense suppression and the treatment of disease 失效
标题翻译：使用乙酰氨基苯甲酸化合物进行无味抑制和治疗疾病
公开(公告)号：US20070203123A1
公开(公告)日：2007-08-30
申请号：US11789625
申请日：2007-04-24
申请人： Richard Wilde , Ellen Welch , James Takasugi , Neil Almstead , Steven Rubenstein , Holger Beckmann
发明人： Richard Wilde , Ellen Welch , James Takasugi , Neil Almstead , Steven Rubenstein , Holger Beckmann
IPC分类号： A61K31/55 , A61K31/445 , A61K31/24 , A61K31/195 , A61K31/277
CPC分类号： C07C235/24 , C07C235/74 , C07C323/60 , C07C327/42 , C07C2601/08 , C07C2602/08 , C07C2602/10 , C07D207/273 , C07D209/48 , C07D213/74 , C07D239/38 , C07D257/04 , C07D271/10 , C07D317/64
摘要： Novel acetylamino benzoic acid compounds, methods of using and pharmaceutical compositions comprising an acetylamino benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.
摘要翻译：公开了新型乙酰氨基苯甲酸化合物，使用方法和包含乙酰氨基苯甲酸衍生物的药物组合物。所述方法包括治疗或预防通过调节过早翻译终止或无义介导的mRNA衰变而改善的疾病或改善与其相关的一种或多种症状的方法。

3. 发明申请

US20060167263A1 Acetylamino benzoic acid compounds and their use for nonsense suppression and the treatment of disease 有权
标题翻译：乙酰氨基苯甲酸化合物及其用于无意义抑制和治疗疾病
公开(公告)号：US20060167263A1
公开(公告)日：2006-07-27
申请号：US11048657
申请日：2005-01-21
申请人： Richard Wilde , Ellen Welch , James Takasugi , Neil Almstead , Steven Rubenstein , Holger Beckmann
发明人： Richard Wilde , Ellen Welch , James Takasugi , Neil Almstead , Steven Rubenstein , Holger Beckmann
IPC分类号： C07D209/46 , C07D207/24 , C07C327/38
CPC分类号： C07C235/24 , C07C235/74 , C07C323/60 , C07C327/42 , C07C2601/08 , C07C2602/08 , C07C2602/10 , C07D207/273 , C07D209/48 , C07D213/74 , C07D239/38 , C07D257/04 , C07D271/10 , C07D317/64
摘要： Novel acetylamino benzoic acid compounds, methods of using and pharmaceutical compositions comprising an acetylamino benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.
摘要翻译：公开了新型乙酰氨基苯甲酸化合物，使用方法和包含乙酰氨基苯甲酸衍生物的药物组合物。所述方法包括治疗或预防通过调节过早翻译终止或无义介导的mRNA衰变而改善的疾病或改善与其相关的一种或多种症状的方法。

4. 发明申请

US20060166926A1 Use of nucleoside compounds for nonsense suppression and the treatment of genetic diseases 有权
标题翻译：使用核苷化合物进行无意义抑制和遗传性疾病的治疗
公开(公告)号：US20060166926A1
公开(公告)日：2006-07-27
申请号：US11048659
申请日：2005-01-21
申请人： Richard Wilde , Neil Almstead , Ellen Welch , Holger Beckmann
发明人： Richard Wilde , Neil Almstead , Ellen Welch , Holger Beckmann
IPC分类号： A61K31/7072 , A61K31/506 , C07D405/02 , C07H19/048
CPC分类号： A61K31/506 , A61K31/7072 , C07H19/00
摘要： The invention encompasses nucleoside compounds, compositions comprising the compounds and methods for treating or preventing diseases associated with nonsense mutations of mRNA by administering these compounds or compositions.
摘要翻译：本发明包括核苷化合物，包含该化合物的组合物和通过施用这些化合物或组合物来治疗或预防与无义突变mRNA相关的疾病的方法。

5. 发明申请

US20080280869A1 Pyrazole or Triazole Compounds and Their Use for the Manufacture of a Medicament for Treating Somatic Mutation-Related Diseases 有权
标题翻译：吡唑或三唑化合物及其用于制造治疗体细胞突变相关疾病的药物的用途
公开(公告)号：US20080280869A1
公开(公告)日：2008-11-13
申请号：US11577177
申请日：2005-10-13
申请人： Neil Almstead , Gary M. Karp , Richard Wilde , Ellen Welch , Jeffrey A. Campbell , Hongyu Ren , Guangming Chen
发明人： Neil Almstead , Gary M. Karp , Richard Wilde , Ellen Welch , Jeffrey A. Campbell , Hongyu Ren , Guangming Chen
IPC分类号： A61K31/4196 , C07D233/88 , C07D249/08 , C07D231/12 , C07D491/04 , C07D271/06 , A61P35/00 , C12N5/06 , A61P37/02 , A61K31/4245 , A61K31/415 , C07D403/10 , A61K31/4168
CPC分类号： C07D271/06 , C07D231/12 , C07D233/64 , C07D249/08 , C07D251/24 , C07D261/08 , C07D263/32 , C07D271/10 , C07D271/107 , C07D277/30 , C07D285/12 , C07D307/68 , C07D333/24 , C07D401/04 , C07D403/04 , C07D405/04 , C07D409/04 , C07D413/04 , C07D413/06 , C07D413/12 , C07D417/04 , C07F9/65306
摘要： The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.
摘要翻译：本发明涉及通过施用本发明的化合物或组合物来治疗或预防与mRNA中的无义突变有关的疾病的方法，化合物和组合物。更具体地，本发明涉及用于抑制与mRNA中的无义突变相关的过早翻译终止的方法，化合物和组合物。

6. 发明授权

US09051342B2 Pyrazole or triazole compounds and their use for the manufacture of a medicament for treating somatic mutation related diseases 有权
标题翻译：吡唑或三唑化合物及其用于制备用于治疗体细胞突变相关疾病的药物的用途
公开(公告)号：US09051342B2
公开(公告)日：2015-06-09
申请号：US11577177
申请日：2005-10-13
申请人： Neil Almstead , Gary M. Karp , Richard Wilde , Ellen Welch , Jeffrey A. Campbell , Hongyu Ren , Guangming Chen
发明人： Neil Almstead , Gary M. Karp , Richard Wilde , Ellen Welch , Jeffrey A. Campbell , Hongyu Ren , Guangming Chen
IPC分类号： A61K31/397 , A61K31/41 , A61K31/415 , C07F9/653 , C07D231/12 , C07D233/64 , C07D249/08 , C07D261/08 , C07D263/32 , C07D271/06 , C07D277/30 , C07D285/12 , C07D307/68 , C07D333/24 , C07D413/12
CPC分类号： C07D271/06 , C07D231/12 , C07D233/64 , C07D249/08 , C07D251/24 , C07D261/08 , C07D263/32 , C07D271/10 , C07D271/107 , C07D277/30 , C07D285/12 , C07D307/68 , C07D333/24 , C07D401/04 , C07D403/04 , C07D405/04 , C07D409/04 , C07D413/04 , C07D413/06 , C07D413/12 , C07D417/04 , C07F9/65306
摘要： The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.
摘要翻译：本发明涉及通过施用本发明的化合物或组合物来治疗或预防与mRNA中的无义突变有关的疾病的方法，化合物和组合物。更具体地，本发明涉及用于抑制与mRNA中的无义突变相关的过早翻译终止的方法，化合物和组合物。

7. 发明申请

US20080119473A1 Compounds for Nonsense Suppression, and Methods for Their Use 有权
标题翻译：无意义抑制的化合物及其使用方法
公开(公告)号：US20080119473A1
公开(公告)日：2008-05-22
申请号：US11577189
申请日：2005-10-13
申请人： Neil Almstead , Gary M. Karp , Richard Wilde , Ellen Welch , Hongyu Ren
发明人： Neil Almstead , Gary M. Karp , Richard Wilde , Ellen Welch , Hongyu Ren
IPC分类号： A61K31/53 , C07D239/42 , A61K31/435
CPC分类号： C07D271/06 , C07D231/12 , C07D233/64 , C07D249/08 , C07D251/24 , C07D261/08 , C07D263/32 , C07D271/10 , C07D271/107 , C07D277/30 , C07D285/12 , C07D307/68 , C07D333/24 , C07D401/04 , C07D403/04 , C07D405/04 , C07D409/04 , C07D413/04 , C07D413/06 , C07D413/12 , C07D417/04 , C07F9/65306
摘要： The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.
摘要翻译：本发明涉及通过施用本发明的化合物或组合物来治疗或预防与mRNA中的无义突变有关的疾病的方法，化合物和组合物。更具体地，本发明涉及用于抑制与mRNA中的无义突变相关的过早翻译终止的方法，化合物和组合物。

8. 发明授权

US09315467B2 Compounds for nonsense suppression, and methods for their use 有权
公开(公告)号：US09315467B2
公开(公告)日：2016-04-19
申请号：US11577189
申请日：2005-10-13
申请人： Neil Almstead , Gary M. Karp , Richard Wilde , Ellen Welch , Hongyu Ren
发明人： Neil Almstead , Gary M. Karp , Richard Wilde , Ellen Welch , Hongyu Ren
IPC分类号： A61K31/53 , C07D239/42 , C07D401/04 , A61K31/435 , C07D231/12 , C07D233/64 , C07D249/08 , C07D261/08 , C07D263/32 , C07D271/06 , C07D277/30 , C07D285/12 , C07D307/68 , C07D333/24 , C07D413/12 , C07F9/653 , C07D403/04 , C07D405/04 , C07D409/04 , C07D413/04
CPC分类号： C07D271/06 , C07D231/12 , C07D233/64 , C07D249/08 , C07D251/24 , C07D261/08 , C07D263/32 , C07D271/10 , C07D271/107 , C07D277/30 , C07D285/12 , C07D307/68 , C07D333/24 , C07D401/04 , C07D403/04 , C07D405/04 , C07D409/04 , C07D413/04 , C07D413/06 , C07D413/12 , C07D417/04 , C07F9/65306
摘要： The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.

9. 发明申请

US20060257866A1 Methods for identifying small molecules that modulate premature translation termination and nonsense mediated mrna decay 有权
标题翻译：用于鉴定调节过早翻译终止和废话介导的mrna衰变的小分子的方法
公开(公告)号：US20060257866A1
公开(公告)日：2006-11-16
申请号：US10521775
申请日：2003-07-24
申请人： Ellen Welch , Neil Almstead , Robert Rando , Mathew Pellegrini
发明人： Ellen Welch , Neil Almstead , Robert Rando , Mathew Pellegrini
IPC分类号： C40B30/06 , C12Q1/68
CPC分类号： B82Y30/00 , G01N33/5308 , G01N2500/04
摘要： The present invention relates to a method for screening and identifying compounds that modulate premature translation termination and/or nonsense-mediated messenger ribonucleic acid (“mRNA”) by interacting with a preselected target ribonucleic acid (“RNA”). In particular, the present invention relates to identifying compounds that bind to regions of the 28S ribosomal RNA (“rRNA”) and analogs thereof. Direct, noncompetitive binding assays are advantageously used to screen libraries of compounds for those that selectively bind to a preselected target RNA. Binding of target RNA molecules to a particular compound is detected using any physical method that measures the altered physical property of the target RNA bound to a compound. The structure of the compound attached to the labeled RNA is also determined. The methods used will depend, in part, on the nature of the library screened. The methods of the present invention provide a simple, sensitive assay for high-throughput screening of libraries of compounds to identify pharmaceutical leads.
摘要翻译：本发明涉及通过与预选的靶核糖核酸（“RNA”）相互作用来筛选和鉴定调节过早翻译终止和/或无义介导的信使核糖核酸（“mRNA”）的化合物的方法。特别地，本发明涉及鉴定结合28S核糖体RNA（“rRNA”）的区域的化合物及其类似物。直接的非竞争性结合测定法有利地用于筛选选择性结合预选靶RNA的化合物文库。使用测量与化合物结合的靶RNA的改变的物理性质的任何物理方法检测靶RNA分子与特定化合物的结合。还测定了附着于标记的RNA的化合物的结构。所使用的方法部分取决于所筛选图书馆的性质。本发明的方法提供了用于识别药物引线的化合物文库的高通量筛选的简单，灵敏的测定。

10. 发明授权

US09289398B2 Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith 有权
标题翻译：从具有无义突变的DNA生产功能性蛋白质的方法以及与其相关的病症的治疗
公开(公告)号：US09289398B2
公开(公告)日：2016-03-22
申请号：US12294952
申请日：2007-03-29
申请人： Neil Almstead , Guangming Chen , Samit Hirawat , Peter Seongwoo Hwang , Gary M. Karp , Langdon Miller , Young-Choon Moon , Hongyu Ren , James J. Takasugi , Ellen M. Welch , Richard G. Wilde , Paul Kennedy
发明人： Neil Almstead , Guangming Chen , Samit Hirawat , Peter Seongwoo Hwang , Gary M. Karp , Langdon Miller , Young-Choon Moon , Hongyu Ren , James J. Takasugi , Ellen M. Welch , Richard G. Wilde , Paul Kennedy
IPC分类号： A61K31/4245 , A61P21/00 , A61K31/00 , A61K31/40 , A61K31/41 , A61K31/4162 , A61K31/44 , A61K31/46 , A61K31/5377 , A61K31/496 , A61K31/454 , A61K31/4709 , A61K31/4439 , A61K31/519 , A61K31/498 , A61K31/53 , A61K31/506 , A61K31/497 , A61K31/501 , A61K31/433 , A61K31/421 , A61K31/415 , A61K31/426 , A61K31/427 , A61K31/428 , A61K31/42 , A61K31/4164 , A61K31/34 , A61K31/381 , A61K31/4196 , A61P11/00 , A61P7/00 , A61P25/00 , A61P13/12 , A61P27/02
CPC分类号： A61K31/4245 , A61K31/00 , A61K31/40 , A61K31/41 , A61K31/4162 , A61K31/44 , A61K31/46
摘要： The present invention relates to functional proteins encoded by nucleic acid sequences comprising a nonsense mutation. The present invention also relates to methods for the production of functional proteins encoded by nucleic acid sequences comprising a nonsense mutation and the use of such proteins for prevention, management and/or treatment of diseases associated with a nonsense mutation(s) in a gene.
摘要翻译：本发明涉及由包含无义突变的核酸序列编码的功能性蛋白质。本发明还涉及用于产生由包含无义突变的核酸序列编码的功能性蛋白质的方法以及用于预防，管理和/或治疗与基因中的无义突变有关的疾病的这些蛋白质的用途。

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