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1. 发明专利

JP2014028816A Method and composition for modulating sialic acid production and treating hereditary inclusion body myopathy 审中-公开
标题翻译：调节酸乳酸生产的方法和组合物，并处理包括身体的肌肉酸痛
公开(公告)号：JP2014028816A
公开(公告)日：2014-02-13
申请号：JP2013171858
申请日：2013-08-22
申请人： Gradalis Inc , グラダリスインク.Gradalis,Inc.
发明人： MAPLES PHILLIP , CHRIS JAY , JOHN NEMUNAITIS
IPC分类号： A61K48/00 , A61K9/08 , A61K9/16 , A61K38/45 , A61K38/52 , A61K47/44 , A61K47/48 , A61P3/00 , A61P21/00 , A61P43/00 , C12N15/09
CPC分类号： A61K48/005 , C12N9/90 , C12N15/85 , C12Y501/03014
摘要： PROBLEM TO BE SOLVED: To provide methods and compositions for modulating sialic acid production in a system, as well as methods and compositions for treating and/or preventing Hereditary Inclusion Body Myopathy and/or symptoms thereof.SOLUTION: Methods for modulating the production of sialic acid in a system are provided, which comprise providing the system with a wild-type GNE-encoding nucleic acid sequence, wherein the system comprises a cell, muscular tissue, or other desirable targets. The present invention encompasses methods for producing wild-type GNE in a system that comprises a mutated endogenous GNE-encoding sequence, which methods comprise providing, for example, a cell or muscular tissue that harbors a mutated (defective) GNE-encoding sequence with a functional wild-type GNE encoding sequence. Also provided is a method for modulating the production of sialic acid in a system, which comprises providing the system with a wild-type GNE-encoding nucleic acid sequence comprising a promoter being the CMV promoter.
摘要翻译：要解决的问题：提供用于调节系统中唾液酸生产的方法和组合物，以及用于治疗和/或预防遗传性包涵体肌病和/或其症状的方法和组合物。解决方案：调节唾液酸生产的方法提供系统中的酸，其包括向系统提供编码野生型GNE的核酸序列，其中所述系统包含细胞，肌肉组织或其它期望的靶标。本发明包括在包含突变的内源性GNE编码序列的系统中产生野生型GNE的方法，所述方法包括提供例如含有突变（缺陷）GNE编码序列的细胞或肌肉组织，其具有功能野生型GNE编码序列。还提供了一种用于调节系统中唾液酸生成的方法，其包括向系统提供包含CMV启动子的启动子的野生型GNE编码核酸序列。

2. 发明申请

WO2008097623A3 METHODS AND COMPOSITIONS FOR MODULATING SIALIC ACID PRODUCTION AND TREATING HEREDITARY INCLUSION BODY MYOPATHY 审中-公开
标题翻译：调节酸乳酸生产和治疗包括身体肌肉酸痛的方法和组合物
公开(公告)号：WO2008097623A3
公开(公告)日：2008-12-31
申请号：PCT/US2008001650
申请日：2008-02-07
申请人： GRADALIS INC , MAPLES PHILLIP , JAY CHRIS , NEMUNAITIS JOHN
发明人： MAPLES PHILLIP , JAY CHRIS , NEMUNAITIS JOHN
IPC分类号： A01N43/04 , A61K31/70 , C07H21/02 , C07H21/04
CPC分类号： A61K48/005 , C12N9/90 , C12N15/85 , C12Y501/03014
摘要： According to certain embodiments of the present invention, methods for modulating the production of sialic acid in a system are provided, which comprise providing the system with a wild-type GNE-encoding nucleic acid sequence. According to such embodiments, the system may comprise a cell, muscular tissue, or other desirable targets. Similarly, the present invention encompasses methods for producing wild-type GNE in a system that comprises a mutated endogenous GNE-encoding sequence. In other words, the present invention includes providing, for example, a cell or muscular tissue that harbors a mutated (defective) GNE-encoding sequence with a functional wild-type GNE encoding sequence.
摘要翻译：根据本发明的某些实施方案，提供了调节系统中唾液酸生产的方法，其包括向系统提供编码野生型GNE的核酸序列。根据这样的实施例，该系统可以包括细胞，肌肉组织或其它期望的靶。类似地，本发明包括在包含突变的内源GNE编码序列的系统中产生野生型GNE的方法。换句话说，本发明包括例如提供具有功能性野生型GNE编码序列的突变（缺陷）GNE编码序列的细胞或肌肉组织。

3. 发明公开

EP2117304A4 METHODS AND COMPOSITIONS FOR MODULATING SIALIC ACID PRODUCTION AND TREATING HEREDITARY INCLUSION BODY MYOPATHY 审中-公开
标题翻译：调制SIALSÄUREPRODUKTION及治疗方法和组合物遗传性包涵体肌病
公开(公告)号：EP2117304A4
公开(公告)日：2011-08-24
申请号：EP08725300
申请日：2008-02-07
申请人： GRADALIS INC
发明人： MAPLES PHILLIP , JAY CHRIS , NEMUNAITIS JOHN
IPC分类号： A01N43/04 , A61K31/70 , A61K48/00 , C07H21/02 , C07H21/04
CPC分类号： A61K48/005 , C12N9/90 , C12N15/85 , C12Y501/03014

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