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公开(公告)号：US11959145B2
公开(公告)日：2024-04-16
申请号：US13320172
申请日：2010-05-12
IPC分类号： C12N15/63 , C12Q1/6883 , C12Q1/6897
CPC分类号： C12Q1/6897 , C12Q1/6883 , C12Q2600/136 , C12Q2600/158
摘要： The present invention relates to systems and methods for screening compounds and/or mutant ribosomal proteins in a eukaryotic cell that increase or decrease the translation of a target gene and thereby ameliorate or revert a defective and/or undesired translation of a target gene. Disclosed are compounds and proteins as identified with the methods and systems of the invention, pharmaceutical and cosmetic compositions thereof, their uses for the preparation of a medicament, methods of treatment of a disease or condition or cosmetic condition related to the defective translation of a gene, for example genetic diseases such as Epidermolysis bullosa, as well as diagnostic measures practical for the clinical evaluation of such diseases or conditions. Also, kits are provided which comprise the identified compounds and/or proteins in addition to suitable means for performing the methods of the invention.
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2.发明申请Method for Identifying Interventions that Control the Translational Activity of Ribosomal Proteins in Different MRNA Expression 审中-公开识别控制不同MRNA表达中核糖体蛋白质转译活性的干预方法公开(公告)号：US20120134983A1
公开(公告)日：2012-05-31
申请号：US13320172
申请日：2010-05-12
IPC分类号： A61K39/395 , A61K8/64 , C07K16/00 , C12N1/19 , C12Q1/68
摘要： The present invention relates to systems and methods for screening compounds and/or mutant ribosomal proteins in a eukaryotic cell that increase or decrease the translation of a target gene and thereby ameliorate or revert a defective and/or undesired translation of a target gene. Disclosed are compounds and proteins as identified with the methods and systems of the invention, pharmaceutical and cosmetic compositions thereof, their uses for the preparation of a medicament, methods of treatment of a disease or condition or cosmetic condition related to the defective translation of a gene, for example genetic diseases such as Epidermolysis bullosa, as well as diagnostic measures practical for the clinical evaluation of such diseases or conditions. Also, kits are provided which comprise the identified compounds and/or proteins in addition to suitable means for performing the methods of the invention
摘要翻译： 本发明涉及用于筛选真核细胞中化合物和/或突变核糖体蛋白的系统和方法，其增加或减少靶基因的翻译，从而改善或恢复靶基因的有缺陷和/或不需要的翻译。 公开了本发明的方法和系统，其药物和化妆品组合物，其用于制备药物的用途，治疗疾病或病症的方法或与基因的有缺陷翻译相关的美容条件的化合物和蛋白质 ，例如遗传性疾病如大疱性表皮松解症，以及用于临床评估这些疾病或病症的诊断措施。 此外，提供了包含鉴定的化合物和/或蛋白质的试剂盒，以及用于进行本发明的方法的合适的方法
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公开(公告)号：US20240124944A1
公开(公告)日：2024-04-18
申请号：US18525193
申请日：2023-11-30
IPC分类号： C12Q1/6897 , C12Q1/6883
CPC分类号： C12Q1/6897 , C12Q1/6883 , C12Q2600/136 , C12Q2600/158
摘要： The present invention relates to systems and methods for screening compounds and/or mutant ribosomal proteins in a eukaryotic cell that increase or decrease the translation of a target gene and thereby ameliorate or revert a defective and/or undesired translation of a target gene. Disclosed are compounds and proteins as identified with the methods and systems of the invention, pharmaceutical and cosmetic compositions thereof, their uses for the preparation of a medicament, methods of treatment of a disease or condition or cosmetic condition related to the defective translation of a gene, for example genetic diseases such as Epidermolysis bullosa, as well as diagnostic measures practical for the clinical evaluation of such diseases or conditions. Also, kits are provided which comprise the identified compounds and/or proteins in addition to suitable means for performing the methods of the invention.
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