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1. 发明授权

US08986935B2 Methods for treating spinal muscular atrophy 有权
标题翻译：治疗脊髓性肌萎缩的方法
公开(公告)号：US08986935B2
公开(公告)日：2015-03-24
申请号：US13058653
申请日：2009-08-13
申请人： Sergey V. Paushkin , Nikolai A. Naryshkin , Ellen Welch
发明人： Sergey V. Paushkin , Nikolai A. Naryshkin , Ellen Welch
IPC分类号： C12Q1/68 , G01N33/567 , C12Q1/02
CPC分类号： A61K31/4035 , C12N7/00 , C12N15/11 , C12N2710/16663 , C12N2720/00063 , C12N2740/11063 , C12N2740/12063 , C12N2740/14063 , C12N2740/15063 , C12N2740/16063 , C12N2770/00063 , C12N2770/10063 , C12N2770/12063 , C12N2770/20063 , C12N2770/38063 , C12N2795/10263 , C12N2795/10363 , C12Q1/66 , C12Q1/6897 , Y02A50/491
摘要： Described herein are methods for the identification or validation of compounds capable of causing ribosomal frameshifting and the use of the compounds identified by the methods described herein to produce a stabilized SMNΔEx7 protein and treat Spinal Muscular Atrophy.
摘要翻译：本文描述了用于鉴定或验证能够引起核糖体移码的化合物的方法，以及通过本文所述的方法鉴定的化合物的用途，以产生稳定的SMN＆Dgr。Ex7蛋白并治疗脊髓性肌肉萎缩。

2. 发明申请

US20110213005A1 METHODS FOR TREATING VIRAL INFECTIONS 有权
标题翻译：治疗病毒感染的方法
公开(公告)号：US20110213005A1
公开(公告)日：2011-09-01
申请号：US13058613
申请日：2009-08-13
申请人： Sergey V. Paushkin , Nikolai A. Naryshkin , Ellen Welch
发明人： Sergey V. Paushkin , Nikolai A. Naryshkin , Ellen Welch
IPC分类号： A61K31/4035 , C07H21/02 , A61P31/12 , C12Q1/68 , C12N5/02 , C12N15/63
CPC分类号： A61K31/4035 , C12N7/00 , C12N15/11 , C12N2710/16663 , C12N2720/00063 , C12N2740/11063 , C12N2740/12063 , C12N2740/14063 , C12N2740/15063 , C12N2740/16063 , C12N2770/00063 , C12N2770/10063 , C12N2770/12063 , C12N2770/20063 , C12N2770/38063 , C12N2795/10263 , C12N2795/10363 , C12Q1/66 , C12Q1/6897 , Y02A50/491
摘要： The present invention relates to compounds that modulate ribosomal frameshifting and nucleic acid constructs for use in methods for identifying or validation such compounds. In particular, the present invention relates to the use of nucleic acid constructs to identify or validate compounds capable of modulating the efficiency of programmed ribosomal frameshifting and the use of said compounds to inhibit the replication or infectivity of viruses that employ programmed ribosomal frameshifting.
摘要翻译：本发明涉及调节核糖体移码和核酸构建体以用于鉴定或验证这些化合物的方法中的化合物。特别地，本发明涉及核酸构建体用于鉴定或验证能够调节编程的核糖体移码效率的化合物的用途，以及使用所述化合物抑制采用程序化的核糖体移码的病毒的复制或感染性。

3. 发明申请

US20110172284A1 METHODS FOR TREATING SPINAL MUSCULAR ATROPHY 有权
标题翻译：方法治疗脊柱肌瘤
公开(公告)号：US20110172284A1
公开(公告)日：2011-07-14
申请号：US13058653
申请日：2009-08-13
申请人： Sergey V. Paushkin , Nikolai A. Naryshkin , Ellen Welch
发明人： Sergey V. Paushkin , Nikolai A. Naryshkin , Ellen Welch
IPC分类号： A61K31/4035 , C12Q1/68 , A61P43/00
CPC分类号： A61K31/4035 , C12N7/00 , C12N15/11 , C12N2710/16663 , C12N2720/00063 , C12N2740/11063 , C12N2740/12063 , C12N2740/14063 , C12N2740/15063 , C12N2740/16063 , C12N2770/00063 , C12N2770/10063 , C12N2770/12063 , C12N2770/20063 , C12N2770/38063 , C12N2795/10263 , C12N2795/10363 , C12Q1/66 , C12Q1/6897 , Y02A50/491
摘要： Described herein are methods for the identification or validation of compounds capable of causing ribosomal frameshifting and the use of said compounds to produce a stabilized SMNΔEx7 protein and treat Spinal Muscular Atrophy.
摘要翻译：本文描述了用于鉴定或验证能够引起核糖体移码的化合物的方法，以及使用所述化合物产生稳定的SMN＆Dgr; Ex7蛋白并治疗脊髓性肌肉萎缩的方法。

4. 发明授权

US08932818B2 Screening assays for compounds that modulate programmed ribosomal frameshifting 有权
标题翻译：调节程序性核糖体移码的化合物的筛选测定
公开(公告)号：US08932818B2
公开(公告)日：2015-01-13
申请号：US13058613
申请日：2009-08-13
申请人： Sergey V. Paushkin , Nikolai A. Naryshkin , Ellen Welch
发明人： Sergey V. Paushkin , Nikolai A. Naryshkin , Ellen Welch
IPC分类号： C12Q1/68 , C12Q1/66 , C12Q1/48 , C12Q1/34 , C12Q1/02
CPC分类号： A61K31/4035 , C12N7/00 , C12N15/11 , C12N2710/16663 , C12N2720/00063 , C12N2740/11063 , C12N2740/12063 , C12N2740/14063 , C12N2740/15063 , C12N2740/16063 , C12N2770/00063 , C12N2770/10063 , C12N2770/12063 , C12N2770/20063 , C12N2770/38063 , C12N2795/10263 , C12N2795/10363 , C12Q1/66 , C12Q1/6897 , Y02A50/491
摘要： The present invention relates to compounds that modulate ribosomal frameshifting and nucleic acid constructs for use in methods for identifying or validation of compounds that modulate ribosomal frameshifting. In particular, the present invention relates to the use of nucleic acid constructs to identify or validate compounds capable of modulating the efficiency of programmed ribosomal frameshifting and the use of compounds that modulate the efficiency of programmed ribosomal frameshifting to inhibit the replication or infectivity of viruses that employ programmed ribosomal frameshifting.
摘要翻译：本发明涉及调节核糖体移码和核酸构建体的化合物，其用于鉴定或验证调节核糖体移码的化合物的方法。特别地，本发明涉及核酸构建体用于鉴定或验证能够调节编程的核糖体移码效率的化合物的用途，以及调节编程的核糖体移码效率以抑制病毒的复制或感染性的化合物的用途，采用编程的核糖体移码。

5. 发明授权

US08633019B2 Methods for treating spinal muscular atrophy 有权
标题翻译：治疗脊髓性肌萎缩的方法
公开(公告)号：US08633019B2
公开(公告)日：2014-01-21
申请号：US12994517
申请日：2009-05-27
申请人： Sergey V. Paushkin , Nikolai Naryshkin , Charles Romfo , Ellen Welch
发明人： Sergey V. Paushkin , Nikolai Naryshkin , Charles Romfo , Ellen Welch
IPC分类号： C12Q1/68 , A01N43/00 , C12N15/00
CPC分类号： C07D498/04 , C07D263/57 , C07D413/04 , C07D413/06 , C07D413/12 , C07D413/14 , C07D491/113 , C07K14/475 , C07K2319/61 , C12N2840/00 , C12Q1/6883 , C12Q2600/136 , C12Q2600/158
摘要： The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.
摘要翻译：本发明提供核酸构建体，用于鉴定和验证增加SMN2外显子7包含在从SMN2基因转录的mRNA中的化合物的方法，提高从SMN2基因产生的SMN蛋白质水平的化合物和药物组合物，以及用于其用于治疗SMA。

6. 发明申请

US20110086833A1 METHODS FOR TREATING SPINAL MUSCULAR ATROPHY 有权
标题翻译：方法治疗脊柱肌瘤
公开(公告)号：US20110086833A1
公开(公告)日：2011-04-14
申请号：US12994517
申请日：2009-05-27
申请人： Sergey V. Paushkin , Nikolai Naryshkin , Charles Romfo , Ellen Welch
发明人： Sergey V. Paushkin , Nikolai Naryshkin , Charles Romfo , Ellen Welch
IPC分类号： A61K31/423 , C07H21/04 , C12N5/10 , C12Q1/68 , A61P25/00 , A61K31/5377 , A61K31/454 , A61K31/438 , A61K31/496 , A61K31/437 , A61K31/4439 , A61K31/551
CPC分类号： C07D498/04 , C07D263/57 , C07D413/04 , C07D413/06 , C07D413/12 , C07D413/14 , C07D491/113 , C07K14/475 , C07K2319/61 , C12N2840/00 , C12Q1/6883 , C12Q2600/136 , C12Q2600/158
摘要： The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.
摘要翻译：本发明提供核酸构建体，用于鉴定和验证增加SMN2外显子7包含在从SMN2基因转录的mRNA中的化合物的方法，提高从SMN2基因产生的SMN蛋白质水平的化合物和药物组合物，以及用于其用于治疗SMA。

7. 发明申请

US20090253699A1 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：US20090253699A1
公开(公告)日：2009-10-08
申请号：US11577191
申请日：2005-10-13
申请人： Neil G. Almstead , Guangming Chen , Gary M. Karp , Ellen Welch , Richard Wilde , Jeffrey A. Campbell
发明人： Neil G. Almstead , Guangming Chen , Gary M. Karp , Ellen Welch , Richard Wilde , Jeffrey A. Campbell
IPC分类号： A61K31/5377 , A61K31/4245 , C07D271/10 , C12N5/00 , C07D413/02 , A61K31/433 , C07D285/12 , A61K31/421 , C07D263/30 , A61K31/415 , C07D231/10 , A61K31/426 , C07D277/20 , C07D307/02 , A61K31/34 , A61P37/06 , A61P7/00 , A61P25/00 , A61P9/00 , A61P29/00
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

US08283115B1 Methods of screening for compounds for treating muscular dystrophy using UTRN mRNA translation regulation 有权
标题翻译：使用UTRN mRNA翻译调节法筛选化合物治疗肌营养不良的方法
公开(公告)号：US08283115B1
公开(公告)日：2012-10-09
申请号：US12143705
申请日：2008-06-20
申请人： Westley J Friesen , Nikolai Naryshkin , Meenal Patel , Charles Romfo , Ellen Welch , Yuki Tomizawa , Jin Zhuo
发明人： Westley J Friesen , Nikolai Naryshkin , Meenal Patel , Charles Romfo , Ellen Welch , Yuki Tomizawa , Jin Zhuo
IPC分类号： C12Q1/68
CPC分类号： C12Q1/6897 , C07K14/70546 , C12N15/1138 , C12N15/8209 , C12Q1/6883 , C12Q2600/136 , G01N33/5023
摘要： The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of muscular dystrophy (MD), or a form thereof, in which said compounds increase the post-transcriptional expression of a target gene (i.e., mIGF1, ITGA7, or UTRN).
摘要翻译：本发明提供用于鉴定和验证用于治疗肌营养不良（MD）或其形式的化合物的化合物和测定法，其中所述化合物增加靶基因的转录后表达（即mIGF1， ITGA7或UTRN）。

9. 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。

10. 发明授权

US07291461B2 Methods for identifying small molecules that modulate premature translation termination and nonsense mRNA decay 有权
标题翻译：用于鉴定调节过早翻译终止和无义mRNA衰变的小分子的方法
公开(公告)号：US07291461B2
公开(公告)日：2007-11-06
申请号：US10519243
申请日：2003-06-23
申请人： Ellen Welch , Jin Zhuo
发明人： Ellen Welch , Jin Zhuo
IPC分类号： C12Q1/68 , C07H21/02 , C07H21/04
CPC分类号： C12Q1/6897
摘要： The present invention relates to methods for identifying compounds that modulate premature translation termination and/or nonsense-mediated mRNA decay by screening and identifying compounds that modulate the post-transcriptional expression of any gene with a premature translation stop codon. The invention particularly relates to using any gene encoding a premature stop codon to identify compounds that modulate premature translation termination and/or nonsense-mediated mRNA decay. A compound that modulates premature translation termination and/or nonsense-mediated mRNA decay of a target gene is identified using standard methods known in the art to measure changes in translation or mRNA stability of the gene product or mRNA of the gene with the premature stop codon. 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：本发明涉及通过筛选和鉴定调节具有过早翻译终止密码子的任何基因的转录后表达的化合物来鉴定调节过早翻译终止和/或无义介导的mRNA衰变的化合物的方法。本发明特别涉及使用编码过早终止密码子的任何基因来鉴定调节过早翻译终止和/或无义介导的mRNA衰变的化合物。使用本领域已知的标准方法鉴定调节前体转录终止和/或无义介导的mRNA基因的衰老的化合物，以测量基因产物或基因mRNA的mRNA稳定性的变化，所述基因产物或mRNA的早期终止密码子。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。

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