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公开(公告)号：US20190248873A1
公开(公告)日：2019-08-15
申请号：US16250314
申请日：2019-01-17
CPC分类号： C07K14/81 , C07K2319/02 , C07K2319/07 , C07K2319/09 , C07K2319/20 , C07K2319/33 , C07K2319/74 , C12N15/52
摘要： Some embodiments of the systems, methods and compositions provided herein relate to a compound protease. In some embodiments, the compound protease includes a protease domain and a cut site for another enzyme. In some embodiments, the compound protease includes an association domain. In some embodiments, the compound protease is part of a protein circuit.
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公开(公告)号：US20180327458A1
公开(公告)日：2018-11-15
申请号：US16043376
申请日：2018-07-24
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , UNIVERSITÉ PAUL SABATIER TOULOUSE III , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) , UNIVERSITÉ PIERRE ET MARIE CURIE (PARIS 6) , ICM (INSTITUT DU CERVEAU ET DE LA MOELLE EPINIERE)
IPC分类号： C07K14/005 , C12N7/00 , A61K38/16
CPC分类号： C07K14/005 , A61K38/162 , C07K2319/07 , C07K2319/10 , C12N7/00 , C12N2740/16322 , C12N2760/00022 , C12N2760/00033
摘要： The present invention relates to methods and compositions for the prevention or treatment of neurodegenerative diseases.
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公开(公告)号：US20170224783A1
公开(公告)日：2017-08-10
申请号：US15429132
申请日：2017-02-09
申请人： Yissum Research Development Company of the Hebrew University of Jerusalem, Ltd. , Hadasit Medical Research Services and Development Ltd.
IPC分类号： A61K38/44
CPC分类号： A61K38/44 , C07K2319/07 , C07K2319/10 , C12N9/0051 , C12N2740/16011 , C12Y108/01004
摘要： The present invention concerns in general novel fusion proteins comprising a membrane transferring moiety and an enzymatic moiety. The present invention further concerns a method of treating disease using said fusion proteins.
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公开(公告)号：US20170016912A1
公开(公告)日：2017-01-19
申请号：US15263182
申请日：2016-09-12
发明人： Angela Huang
IPC分类号： G01N33/68 , C07K14/705 , G01N33/58 , C12N15/85
CPC分类号： C07K14/705 , C07K14/43595 , C07K14/4716 , C07K14/4728 , C07K2319/03 , C07K2319/04 , C07K2319/07 , C07K2319/09 , C07K2319/60 , G01N2500/04 , G01N2500/10
摘要： This invention provides biosensors, cell models, and methods of their use for monitoring heme, oxygen or ATP. Biosensors can include targeting domains, sensing domains and reporting domains. Biosensors can be introduced into cells reprogrammed to represent experimental or pathologic cells of interest. Model cells expressing the biosensors can be contacted with putative bioactive agents to determine possible activities.
摘要翻译： 本发明提供生物传感器，细胞模型及其用于监测血红素，氧或ATP的方法。 生物传感器可以包括定位域，感知域和报告域。 可以将生物传感器引入重新编程以代表感兴趣的实验或病理细胞的细胞中。 可以将表达生物传感器的模型细胞与推定的生物活性剂接触以确定可能的活性。
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公开(公告)号：US09528999B1
公开(公告)日：2016-12-27
申请号：US15263182
申请日：2016-09-12
发明人： Angela Huang
IPC分类号： G01N33/58 , C07K14/705 , G01N33/68 , C12N15/85
CPC分类号： C07K14/705 , C07K14/43595 , C07K14/4716 , C07K14/4728 , C07K2319/03 , C07K2319/04 , C07K2319/07 , C07K2319/09 , C07K2319/60 , G01N2500/04 , G01N2500/10
摘要： This invention provides biosensors, cell models, and methods of their use for monitoring heme, oxygen or ATP. Biosensors can include targeting domains, sensing domains and reporting domains. Biosensors can be introduced into cells reprogrammed to represent experimental or pathologic cells of interest. Model cells expressing the biosensors can be contacted with putative bioactive agents to determine possible activities.
摘要翻译： 本发明提供生物传感器，细胞模型及其用于监测血红素，氧或ATP的方法。 生物传感器可以包括定位域，感知域和报告域。 可以将生物传感器引入重新编程以代表感兴趣的实验或病理细胞的细胞中。 可以将表达生物传感器的模型细胞与推定的生物活性剂接触以确定可能的活性。
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公开(公告)号：US09017999B2
公开(公告)日：2015-04-28
申请号：US11913618
申请日：2006-05-03
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： The inventors demonstrate that mRNA sorting to the mitochondrial surface is an efficient way to proceed to such an allotopic expression, and that this mRNA sorting can be controlled by selecting appropriate mitochondrion-targeting sequence (MTS) and appropriate 3′UTR sequences. The CDS sequence which codes for the protein to be delivered into the mitochondrion is guided by these appropriate MTS and 3′UTR sequences from the nuclear compartment to the mitochondrion-bound polysomes (where the CDS is translated), and aids in an efficient translocation of a mature functional protein into the mitochondria. Appropriate MTS and 3′UTR sequences correspond to those of nuclearly-transcribed mitochondrially-targeted mRNAs. The inventors demonstrate that, to obtain a stable therapeutically-effective importation, both an appropriate MTS and an appropriate 3′UTR should preferably be used.
摘要翻译： 本发明人证明，对线粒体表面的mRNA分选是进行这种同种异体表达的有效方式，并且可以通过选择适当的线粒体靶向序列（MTS）和合适的3'UTR序列来控制该mRNA分选。 编码要递送到线粒体中的蛋白质的CDS序列由这些适当的MTS和3'UTR序列从核隔室引导到线粒体结合的多核糖体（其中CDS被翻译），并且有助于有效移位 成熟的功能蛋白进入线粒体。 适当的MTS和3'UTR序列对应于核转录的线粒体靶向mRNA的那些。 发明人证明，为了获得稳定的治疗有效的进口，优选使用适当的MTS和适当的3'UTR。
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公开(公告)号：US08952133B2
公开(公告)日：2015-02-10
申请号：US13399434
申请日：2012-02-17
申请人： Shaharyar Khan
发明人： Shaharyar Khan
IPC分类号： C07K14/00 , C07K14/435 , C12Q1/68
CPC分类号： C12N15/87 , A61K48/00 , A61K48/005 , A61K48/0058 , C07K2319/035 , C07K2319/07 , C07K2319/09 , C07K2319/10 , C07K2319/81 , C12N15/62 , C12N15/85 , C12N2810/40
摘要： Methods and compositions for delivering polynucleotides are provided. One embodiment provides a non-viral vector comprising a recombinant polynucleotide-binding protein comprising a protein transduction domain operably linked to a targeting signal.
摘要翻译： 提供了用于递送多核苷酸的方法和组合物。 一个实施方案提供了包含重组多核苷酸结合蛋白的非病毒载体，所述重组多核苷酸结合蛋白包含与靶向信号可操作地连接的蛋白转导结构域。
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8.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：US08865160B2
公开(公告)日：2014-10-21
申请号：US12866540
申请日：2009-02-09
IPC分类号： A61K38/54 , A61P11/00 , A61P1/00 , C12N5/0781 , C12N9/22 , A61K47/48 , C12N9/88 , C12N9/24 , A61K38/00
CPC分类号： C12N9/2497 , A61K38/00 , A61K47/62 , C07K14/005 , C07K14/435 , C07K2319/07 , C07K2319/10 , C12N7/00 , C12N9/22 , C12N9/88 , C12N2740/16371 , C12Y301/21005 , C12Y302/02023 , C12Y402/99018
摘要： The present invention is directed to compositions and methods of preserving viability of islets of Langerhans for transplantation, and treating various diseases and other abnormal or pathological conditions, including inflammatory bowel disease, ischemic heart disease, acute lung injury, acute respiratory distress syndrome and radiation-induced brain injury, with DNA repair enzymes that are directed to the mitochondria.
摘要翻译： 本发明涉及保存用于移植的朗格汉斯胰岛的存活力的组合物和方法，以及治疗各种疾病和其他异常或病理状况，包括炎性肠病，缺血性心脏病，急性肺损伤，急性呼吸窘迫综合征和放射线 - 诱导脑损伤，DNA修复酶被导向线粒体。
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公开(公告)号：US20140308262A1
公开(公告)日：2014-10-16
申请号：US14034224
申请日：2013-09-23
申请人： BioBlast Pharma Ltd.
IPC分类号： C12N9/02 , C07K14/005
CPC分类号： C07K14/47 , A61K38/1709 , C07K14/005 , C07K14/435 , C07K2319/01 , C07K2319/07 , C07K2319/10 , C07K2319/21 , C12N9/0091 , C12N9/1018 , Y10S530/826 , Y10S530/827
摘要： The invention provides fusion protein constructs comprising a functional mitochondrial protein and methods of treating mitochondrial disorders by the fusion proteins and compositions thereof.
摘要翻译： 本发明提供包含功能性线粒体蛋白的融合蛋白构建体和通过融合蛋白及其组合物治疗线粒体病症的方法。
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