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公开(公告)号：US20240093229A1
公开(公告)日：2024-03-21
申请号：US18325321
申请日：2023-05-30
CPC分类号： C12N15/85 , A61K48/005 , A61K48/0058 , A61K48/0066 , C07K14/47 , C12N9/0053 , C12N9/0089 , C12N9/1085 , C12N15/8509 , C12Y109/03001 , C07K2319/07 , C12N2800/22 , C12N2810/80
摘要： An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3′UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3′UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.
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公开(公告)号：US20160256571A1
公开(公告)日：2016-09-08
申请号：US14389368
申请日：2013-09-30
申请人： SANOFI , UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (C.N.R.S.) , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
CPC分类号： A61K48/0066 , A61K38/1722 , A61K38/42 , A61K48/005 , C12N15/113 , C12N2310/14 , C12N2750/14143 , C12N2750/14171
摘要： The present invention concerns a neuroglobin agonist for use in the treatment or prevention of a mitochondrial disease associated with respiratory chain complex I (RCCI) deficiency and/or respiratory chain complex III (RCCIII) deficiency.
摘要翻译： 本发明涉及用于治疗或预防与呼吸链复合体I（RCCI）缺乏和/或呼吸链复合体III（RCCIII）缺陷相关的线粒体疾病的神经球蛋白激动剂。
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公开(公告)号：US20230220414A1
公开(公告)日：2023-07-13
申请号：US17934981
申请日：2022-09-23
CPC分类号： C12N15/85 , C12N9/0089 , A61K48/0058 , A61K48/0066 , C07K14/47 , C12N9/0053 , C12N15/8509 , A61K48/005 , C12Y109/03001 , C12N9/1085 , C12N2810/80 , C12N2800/22 , C07K2319/07
摘要： An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3′UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3′UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.
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公开(公告)号：US20150087054A1
公开(公告)日：2015-03-26
申请号：US14323393
申请日：2014-07-03
CPC分类号： C12N15/85 , A61K48/005 , A61K48/0058 , A61K48/0066 , C07K14/47 , C07K2319/07 , C12N9/0053 , C12N9/0089 , C12N9/1085 , C12N15/8509 , C12N2800/22 , C12N2810/80 , C12Y109/03001
摘要： An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3′UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3′UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.
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5.发明申请公开(公告)号：US20140377869A1
公开(公告)日：2014-12-25
申请号：US14323240
申请日：2014-07-03
CPC分类号： C12N15/85 , A61K48/005 , A61K48/0058 , A61K48/0066 , C07K14/47 , C07K2319/07 , C12N9/0053 , C12N9/0089 , C12N9/1085 , C12N15/8509 , C12N2800/22 , C12N2810/80 , C12Y109/03001
摘要： An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3′UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3′UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.
摘要翻译： 包含适当的线粒体靶向序列（MTS）和适当的3'UTR序列的表达载体提供编码蛋白质（CDS）的mRNA到哺乳动物细胞的线粒体的有效且稳定的递送。 MTS和3'UTR序列将CDS mRNA从细胞的核腔引导到线粒体结合的多核糖体，其中CDS被翻译。 这提供了成熟功能蛋白质有效转运到线粒体中。 还提供了将在哺乳动物细胞的核隔室中表达的mRNA靶向线粒体的方法。 载体和方法可用于治疗线粒体功能缺陷。
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公开(公告)号：US20220389446A1
公开(公告)日：2022-12-08
申请号：US17746181
申请日：2022-05-17
摘要： An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3′UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3′UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.
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公开(公告)号：US20200277623A1
公开(公告)日：2020-09-03
申请号：US16574685
申请日：2019-09-18
摘要： An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3′UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3′UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.
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8.发明申请公开(公告)号：US20150225740A1
公开(公告)日：2015-08-13
申请号：US14676846
申请日：2015-04-02
IPC分类号： C12N15/85
CPC分类号： C12N15/85 , A61K48/005 , A61K48/0058 , A61K48/0066 , C07K14/47 , C07K2319/07 , C12N9/0053 , C12N9/0089 , C12N9/1085 , C12N15/8509 , C12N2800/22 , C12N2810/80 , C12Y109/03001
摘要： An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3′UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3′UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.
摘要翻译： 包含适当的线粒体靶向序列（MTS）和适当的3'UTR序列的表达载体提供编码蛋白质（CDS）的mRNA到哺乳动物细胞的线粒体的有效且稳定的递送。 MTS和3'UTR序列将CDS mRNA从细胞的核腔引导到线粒体结合的多核糖体，其中CDS被翻译。 这提供了成熟功能蛋白质有效转运到线粒体中。 还提供了将在哺乳动物细胞的核隔室中表达的mRNA靶向线粒体的方法。 载体和方法可用于治疗线粒体功能缺陷。
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