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公开(公告)号：US20180256752A1
公开(公告)日：2018-09-13
申请号：US15988728
申请日：2018-05-24
CPC分类号： A61K48/0066 , A61K38/465 , A61K38/4873 , A61K48/0058 , A61K48/0075 , C07K14/4707 , C12N9/16 , C12N9/6472 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12N2830/008 , C12Y301/03048 , C12Y304/22054
摘要： The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
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公开(公告)号：US20230277686A1
公开(公告)日：2023-09-07
申请号：US18004372
申请日：2021-07-09
申请人： GENETHON , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCH MEDICALE) , UNIVERSITE D'EVRY-VAL-D'ESSONNE
发明人： William LOSTAL , Isabelle RICHARD
CPC分类号： A61K48/0058 , C12N15/86 , C12N9/6472 , C12Y304/22054 , C07K14/4708 , C07K14/47 , C07K14/471 , A61P21/00 , C12N2750/14143 , C12N2820/007 , C07K2319/00
摘要： The present invention concerns a novel short promoter characterized by a high activity in the skeletal muscles and a low activity in the heart. It then constitutes a valuable candidate especially for driving the expression of transgenes encoding proteins useful for the treatment of muscular dystrophies.
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公开(公告)号：US20160058890A1
公开(公告)日：2016-03-03
申请号：US14782396
申请日：2014-04-10
CPC分类号： A61K48/0066 , A61K38/465 , A61K38/4873 , A61K48/0058 , A61K48/0075 , C07K14/4707 , C12N9/16 , C12N9/6472 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12N2830/008 , C12Y301/03048 , C12Y304/22054
摘要： The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles and/or the peripheral nervous tissue; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
摘要翻译： 本发明涉及全身给药的表达系统，其包含编码蛋白质的序列，所述表达系统允许：包括骨骼肌和/或周围神经组织在内的靶组织中蛋白质的治疗可接受水平的表达; 以及在靶组织以外的组织中，特别是心脏中的蛋白质在毒性可接受水平的表达。
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公开(公告)号：US11819478B2
公开(公告)日：2023-11-21
申请号：US15988728
申请日：2018-05-24
CPC分类号： A61K48/0066 , A61K38/465 , A61K38/4873 , A61K48/0058 , A61K48/0075 , C12N9/16 , C12N9/6472 , C12N15/86 , C07K14/4707 , C12N2750/14132 , C12N2750/14143 , C12N2830/008 , C12Y301/03048 , C12Y304/22054
摘要： The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing:
the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles; and
the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.-
公开(公告)号：US09981049B2
公开(公告)日：2018-05-29
申请号：US14782396
申请日：2014-04-10
CPC分类号： A61K48/0066 , A61K38/465 , A61K38/4873 , A61K48/0058 , A61K48/0075 , C07K14/4707 , C12N9/16 , C12N9/6472 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12N2830/008 , C12Y301/03048 , C12Y304/22054
摘要： The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles and/or the peripheral nervous tissue; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
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公开(公告)号：US20070093513A1
公开(公告)日：2007-04-26
申请号：US10553320
申请日：2004-04-16
IPC分类号： A61K31/517 , A61K31/454
CPC分类号： A61K38/45 , A61K31/454 , A61K31/517 , C12Y304/22054
摘要： The present invention provides a novel pharmaceutical agent for recovering corneal sensitivity after corneal surgery and improving symptoms of dry eye. This pharmaceutical agent is useful for improving decreased corneal sensitivity and dry eye associated with corneal neurodegeneration such as post-cataract operation, post-LASIK operation, post-PRK operation, postkeratoplasty operation, neuroparalytic keratopathy, corneal ulcer, diabetic keratopathy and the like, since it contains a Rho protein inhibitor.
摘要翻译： 本发明提供了用于在角膜手术后恢复角膜敏感性并改善干眼症状的新型药剂。 该药剂可用于改善与角膜神经变性相关的角膜敏感性和干眼症，如白内障术后，LASIK术后，PRK后术后，角膜内成形术，神经麻痹性角膜病，角膜溃疡，糖尿病性角膜病等。 它含有Rho蛋白抑制剂。
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