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公开(公告)号：US08633019B2
公开(公告)日：2014-01-21
申请号：US12994517
申请日：2009-05-27
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。
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公开(公告)号：US20110086833A1
公开(公告)日：2011-04-14
申请号：US12994517
申请日：2009-05-27
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。
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公开(公告)号：US20110172284A1
公开(公告)日：2011-07-14
申请号：US13058653
申请日：2009-08-13
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。
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公开(公告)号：US08986935B2
公开(公告)日：2015-03-24
申请号：US13058653
申请日：2009-08-13
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。
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公开(公告)号：US20110213005A1
公开(公告)日：2011-09-01
申请号：US13058613
申请日：2009-08-13
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。
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6.发明授权Screening assays for compounds that modulate programmed ribosomal frameshifting 有权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
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。
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7.发明授权Methods of screening for compounds for treating spinal muscular atrophy using SMN mRNA translation regulation 有权使用SMN mRNA翻译调节法筛选用于治疗脊髓性肌萎缩的化合物的方法公开(公告)号：US08283116B1
公开(公告)日：2012-10-09
申请号：US12144577
申请日：2008-06-23
IPC分类号： C12Q1/68
CPC分类号： C12N15/113 , C07K14/4707 , C07K14/705 , C12N5/16 , C12N15/1138 , C12N15/79 , C12N2830/50 , C12Q1/6876 , C12Q1/6897 , C12Q2600/136
摘要： The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of spinal muscular atrophy (SMA), in which said compounds up-regulate the post-transcriptional expression of SMN1 or SMN2.
摘要翻译： 本发明提供用于鉴定和验证用于治疗脊髓性肌萎缩（SMA）的化合物的化合物和测定法，其中所述化合物上调SMN1或SMN2的转录后表达。
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