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公开(公告)号:US20140227300A1
公开(公告)日:2014-08-14
申请号:US14129703
申请日:2012-06-27
申请人: Cynthia Chin , Anthony D. Person , Gary A. Dahl
发明人: Cynthia Chin , Anthony D. Person , Gary A. Dahl
IPC分类号: C12N5/078 , C12N5/074 , C12N5/077 , A61K31/711
CPC分类号: C12N5/0634 , A61K31/7105 , A61K31/711 , A61K38/162 , A61K38/1774 , A61K38/21 , C07K14/005 , C07K14/70503 , C07K14/7156 , C12N5/0658 , C12N5/0696 , C12N2710/20022 , C12N2710/22022 , C12N2710/24122 , C12N2760/16122 , A61K2300/00
摘要: The present invention provides methods, kits, and compositions for reducing an innate immune system response in a human or animal cell, tissue or organism. One embodiment comprises: introducing an Agent mRNA comprising in vitro-synthesized mRNA encoding one or more proteins that affect the induction, activity or response of an innate immune response pathway; whereby, the innate immune response in the cell, tissue or organism is reduced compared to the innate immune response in the absence of the Agent mRNA. Other embodiments are methods, compositions and kits for using an Agent mRNA for treating a disease or medical condition in a human or animal that exhibits symptoms of an elevated innate immune system, or for reducing an innate immune response that is induced in a human or animal cell, tissue or organism by a Foreign Substance that is administered to the cell, tissue or organism.
摘要翻译: 本发明提供用于减少人或动物细胞,组织或生物体内的先天免疫系统应答的方法,试剂盒和组合物。 一个实施方案包括:引入包含影响先天免疫应答途径的诱导,活性或应答的一种或多种蛋白质的体外合成mRNA的试剂mRNA; 由此,在不存在Agent mRNA的情况下,与先天免疫应答相比,细胞,组织或生物体中的先天免疫应答降低。 其它实施方案是使用试剂mRNA用于治疗显示出先天免疫系统升高的症状的人或动物中的疾病或医学病症的方法,组合物和试剂盒,或用于减少在人或动物中诱导的先天免疫应答 细胞,组织或生物体通过给予细胞,组织或生物体的外源物质。
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公开(公告)号:US20240181005A1
公开(公告)日:2024-06-06
申请号:US17998752
申请日:2021-05-17
申请人: CellScript, LLC , THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVIC
发明人: Ronald Meis , Gary Dahl , Suk See De Ravin , Julie Brault , Colin L. Sweeney , Harry L. Malech
IPC分类号: A61K38/17 , A61K31/4184 , A61K31/428 , A61K31/44 , A61K31/7105 , A61K35/17 , A61K35/28 , A61K35/545 , A61K38/46 , A61K48/00 , A61P37/04 , C12N9/22 , C12N15/11 , C12N15/86 , C12N15/90
CPC分类号: A61K38/177 , A61K31/4184 , A61K31/428 , A61K31/44 , A61K31/7105 , A61K35/17 , A61K35/28 , A61K35/545 , A61K38/465 , A61K48/0033 , A61P37/04 , C12N9/22 , C12N15/11 , C12N15/86 , C12N15/907 , C12N2310/20 , C12N2750/14143 , C12N2800/80
摘要: The present invention relates to compositions, systems, and methods for editing a disease/condition causing mutation region in a target gene in a cell. In certain embodiments, the following components are employed: i) mRNA encoding a Tumor Protein p53 (TP53) inhibitor, ii) an inhibiting agent that inhibits Tumor Suppressor p53-Binding Protein 1 (53BPI) (e.g., small molecule EoHR or mRNA encoding a protein that inhibits 53BPI), iii) mRNA encoding a Cas nuclease for CRISPR; iv) a guide RNA specific for a target cleavage site proximal to said disease/condition-causing mutation region; and v) a repair template comprising a region of interest configured to replace said disease/condition-causing mutation region in the target gene during homology-directed repair (HDR). In certain embodiments, the cell is a T-cell, stem cell (e.g., hematopoietic stem cell), or progenitor cell from a subject with the disease or condition (e.g., a Primary Immunodeficiency Disease (PID)). In some embodiments, the gene-edited cell is administered to the subject.
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公开(公告)号:US09005930B2
公开(公告)日:2015-04-14
申请号:US14471649
申请日:2014-08-28
申请人: Cellscript, LLC
发明人: Jerome Jendrisak , Ronald Meis , Gary Dahl
CPC分类号: C12P19/34 , C12N9/1007 , C12N9/1241 , C12N15/1072 , C12N15/1075 , C12N15/1096 , C12N15/111 , C12N15/113 , C12N2310/317 , C12N2320/51 , C12Y201/01056 , C12Y201/01057 , C12Y207/07019 , C12Y207/0705
摘要: The present invention relates to kits and methods for efficiently generating 5′ capped RNA having a modified cap nucleotide and for use of such modified-nucleotide-capped RNA molecules. In particular, the present invention provides kits and methods for capping RNA using a modified cap nucleotide and a capping enzyme system, such as poxvirus capping enzyme. The present invention finds use for in vitro production of 5′-capped RNA having a modified cap nucleotide and for in vitro or in vivo production of polypeptides by in vitro or in vivo translation of such modified-nucleotide-capped RNA. The invention also provides methods and kits for capturing or isolating uncapped RNA comprising primary RNA transcripts or RNA having a 5′-diphosphate, and methods and kits for using a capping enzyme system and modified cap nucleotides for labeling uncapped RNA comprising primary RNA transcripts or RNA having a 5′-diphosphate with detectable dye or enzyme moieties.
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公开(公告)号:US20140221248A1
公开(公告)日:2014-08-07
申请号:US14185384
申请日:2014-02-20
申请人: CELLSCRIPT, LLC
发明人: Jerome Jendrisak , Ronald Meis , Gary Dahl
IPC分类号: C12N15/10
CPC分类号: C12P19/34 , C12N9/1007 , C12N9/1241 , C12N15/1072 , C12N15/1075 , C12N15/1096 , C12N15/111 , C12N15/113 , C12N2310/317 , C12N2320/51 , C12Y201/01056 , C12Y201/01057 , C12Y207/07019 , C12Y207/0705
摘要: The present invention relates to kits and methods for efficiently generating 5′ capped RNA having a modified cap nucleotide and for use of such modified-nucleotide-capped RNA molecules. In particular, the present invention provides kits and methods for capping RNA using a modified cap nucleotide and a capping enzyme system, such as poxvirus capping enzyme. The present invention finds use for in vitro production of 5′-capped RNA having a modified cap nucleotide and for in vitro or in vivo production of polypeptides by in vitro or in vivo translation of such modified-nucleotide-capped RNA. The invention also provides methods and kits for capturing or isolating uncapped RNA comprising primary RNA transcripts or RNA having a 5′-diphosphate, and methods and kits for using a capping enzyme system and modified cap nucleotides for labeling uncapped RNA comprising primary RNA transcripts or RNA having a 5′-diphosphate with detectable dye or enzyme moieties.
摘要翻译: 本发明涉及用于有效产生具有修饰的帽核苷酸的5'封端RNA并用于这种经修饰的核苷酸封端的RNA分子的试剂盒和方法。 特别地,本发明提供了使用修饰的帽核苷酸和封端酶系统(例如痘病毒覆盖酶)对RNA进行封端的试剂盒和方法。 本发明用于通过体外或体内翻译这些经修饰的核苷酸加帽的RNA来体外生产具有修饰的帽核苷酸的5'-加帽的RNA,以及体外或体内产生多肽。 本发明还提供用于捕获或分离包含初级RNA转录物或具有5'-二磷酸的RNA的未封端RNA的方法和试剂盒,以及使用封端酶系统和修饰的帽核苷酸用于标记包含初级RNA转录物或RNA的未加帽RNA的方法和试剂盒 具有可检测的染料或酶部分的5'-二磷酸酯。
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公开(公告)号:US09017970B2
公开(公告)日:2015-04-28
申请号:US12990750
申请日:2009-05-04
CPC分类号: C12Q1/42 , C12N9/14 , C12P19/34 , Y10T436/143333
摘要: The present invention relates to the discovery of RNA 5′ polyphosphatase enzymes not previously described in the art, methods for discovery of said enzymes, compositions of said enzymes, methods for making said enzymes, and various methods and kits for using said enzymes for biomedical research, for human and non-human diagnostics, for production of therapeutic products, and for other applications. In particular, some embodiments provide compositions, kits and methods for employing RNA polyphosphatases for isolation, purification, production, and assay of capped RNA using a biological sample or a sample from an in vitro capping reaction wherein the sample also contains RNA that is not capped. Other embodiments provide compositions, kits and methods wherein RNA polyphosphatases comprise signal-amplifying enzymes for analyte-specific assays.
摘要翻译: 本发明涉及本领域先前未描述的RNA 5'多磷酸酶的发现,所述酶的发现方法,所述酶的组合物,所述酶的制备方法以及使用所述酶用于生物医学研究的各种方法和试剂盒 ,用于人和非人诊断,用于生产治疗产品和其他应用。 具体地,一些实施方案提供了使用RNA多磷酸酶用于使用生物样品或来自体外封端反应的样品分离,纯化,生产和测定加帽RNA的组合物,试剂盒和方法,其中样品还含有未被封端的RNA 。 其他实施方案提供组合物,试剂盒和方法,其中RNA多磷酸酶包含用于分析物特异性测定的信号放大酶。
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公开(公告)号:US08808982B2
公开(公告)日:2014-08-19
申请号:US12962498
申请日:2010-12-07
申请人: Gary Dahl , Anthony Person , Judith Meis , Jerome Jendrisak
发明人: Gary Dahl , Anthony Person , Judith Meis , Jerome Jendrisak
CPC分类号: C12N5/0696 , A61K38/1709 , A61K38/1816 , A61K38/44 , A61K38/465 , A61K38/50 , A61K48/005 , A61K48/0075 , C07K14/47 , C07K14/4712 , C07K14/505 , C12N9/0075 , C12N9/22 , C12N9/78 , C12N15/117 , C12N15/85 , C12N15/87 , C12N2310/17 , C12N2310/335 , C12N2320/30 , C12N2501/602 , C12N2501/603 , C12N2501/604 , C12N2501/605 , C12N2501/606 , C12N2501/608 , C12N2506/02 , C12Y114/13039 , C12Y301/04012 , C12Y305/04004
摘要: The present invention relates to methods for changing the state of differentiation of a eukaryotic cell, the methods comprising introducing mRNA encoding one or more reprogramming factors into a cell and maintaining the cell under conditions wherein the cell is viable and the mRNA that is introduced into the cell is expressed in sufficient amount and for sufficient time to generate a cell that exhibits a changed state of differentiation compared to the cell into which the mRNA was introduced, and compositions therefor. For example, the present invention provides mRNA molecules and methods for their use to reprogram human somatic cells into pluripotent stem cells.
摘要翻译: 本发明涉及用于改变真核细胞分化状态的方法,所述方法包括将编码一种或多种重编程因子的mRNA引入细胞并将细胞维持在其中细胞存活的条件下,并将导入 表达足够量的细胞并足够的时间产生与引入mRNA的细胞相比表现出改变的分化状态的细胞及其组合物。 例如,本发明提供mRNA分子及其用于将人体细胞重编程为多能干细胞的方法。
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公开(公告)号:US11135314B2
公开(公告)日:2021-10-05
申请号:US16225489
申请日:2018-12-19
申请人: CELLSCRIPT, LLC
发明人: Judith Meis , Anthony Person , Cynthia Chin , Jerome Jendrisak , Gary Dahl
IPC分类号: A61K48/00 , A61K38/00 , A61K31/7105 , A61K33/06 , C12N15/10 , C12P21/02 , C12P19/34 , C12N5/074 , C12N5/0793 , C12N5/077 , C12N15/87 , C12P19/38
摘要: The present invention relates to compositions, kits and methods for making and using RNA compositions comprising in vitro-synthesized ssRNA inducing a biological or biochemical effect in a mammalian cell or organism into which the RNA composition is repeatedly or continuously introduced. In certain embodiments, the invention provides compositions and methods for changing the state of differentiation or phenotype of a human or other vertebrate cell. For example, the present invention provides mRNA and methods for reprogramming cells that exhibit a first differentiated state or phenotype to cells that exhibit a second differentiated state or phenotype, such as to reprogram human somatic cells to pluripotent stem cells.
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公开(公告)号:US20210000926A1
公开(公告)日:2021-01-07
申请号:US16968792
申请日:2019-02-19
申请人: The United States of America, as represented by the Secretary, Department of Health and Human Servic , CELLSCRIPT, LLC
发明人: Suk See De Ravin , Harry L. Malech , Ron Meis , Gary A. Dahl
IPC分类号: A61K38/44 , A61K35/17 , A61K35/15 , C12N9/02 , C12N5/0787 , C12N5/0783 , A61P31/00 , A61K35/28 , A61K45/06
摘要: Provided are compositions and methods for treating a subject having a primary immune deficiency (PID), for example who is suffering from a chronic viral, bacterial, or fungal infection, using autologous granulocytes, autologous lymphocytes, and/or NK cells containing exogenous mRNA encoding the missing or defective protein.
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公开(公告)号:US09862926B2
公开(公告)日:2018-01-09
申请号:US14129703
申请日:2012-06-27
申请人: Cynthia Chin , Anthony D. Person , Gary A. Dahl
发明人: Cynthia Chin , Anthony D. Person , Gary A. Dahl
IPC分类号: C12P21/02 , C12N5/078 , A61K38/16 , C07K14/705 , C07K14/715 , A61K31/7105 , A61K38/17 , A61K38/21 , C07K14/005 , A61K31/711 , C12N5/077 , C12N5/074 , A61K48/00 , C07H21/02 , C12P19/34
CPC分类号: C12N5/0634 , A61K31/7105 , A61K31/711 , A61K38/162 , A61K38/1774 , A61K38/21 , C07K14/005 , C07K14/70503 , C07K14/7156 , C12N5/0658 , C12N5/0696 , C12N2710/20022 , C12N2710/22022 , C12N2710/24122 , C12N2760/16122 , A61K2300/00
摘要: The present invention provides methods, kits, and compositions for reducing an innate immune system response in a human or animal cell, tissue or organism. One embodiment comprises: introducing an Agent mRNA comprising in vitro-synthesized mRNA encoding one or more proteins that affect the induction, activity or response of an innate immune response pathway; whereby, the innate immune response in the cell, tissue or organism is reduced compared to the innate immune response in the absence of the Agent mRNA. Other embodiments are methods, compositions and kits for using an Agent mRNA for treating a disease or medical condition in a human or animal that exhibits symptoms of an elevated innate immune system, or for reducing an innate immune response that is induced in a human or animal cell, tissue or organism by a Foreign Substance that is administered to the cell, tissue or organism.
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公开(公告)号:US12059479B2
公开(公告)日:2024-08-13
申请号:US17463007
申请日:2021-08-31
申请人: CELLSCRIPT, LLC
发明人: Judith Meis , Anthony Person , Cynthia Chin , Jerome Jendrisak , Gary Dahl
IPC分类号: A61K48/00 , A61K31/7105 , A61K33/06 , A61K38/00 , C12N5/074 , C12N5/077 , C12N5/0793 , C12N15/10 , C12N15/87 , C12P19/34 , C12P19/38 , C12P21/02
CPC分类号: A61K48/0066 , A61K31/7105 , A61K33/06 , A61K38/005 , A61K48/0041 , C12N5/0619 , C12N5/0658 , C12N5/0696 , C12N15/1003 , C12N15/1096 , C12N15/87 , C12P19/34 , C12P19/38 , C12P21/02 , C12N2501/60 , C12N2501/602 , C12N2501/603 , C12N2501/604 , C12N2501/605 , C12N2501/606 , C12N2501/608 , C12N2501/73 , C12N2506/1307 , C12N2510/00 , C12Y301/26003 , A61K33/06 , A61K2300/00 , A61K31/7105 , A61K2300/00
摘要: The present invention relates to compositions, kits and methods for making and using RNA compositions comprising in vitro-synthesized ssRNA inducing a biological or biochemical effect in a mammalian cell or organism into which the RNA composition is repeatedly or continuously introduced. In certain embodiments, the invention provides compositions and methods for changing the state of differentiation or phenotype of a human or other vertebrate cell. For example, the present invention provides mRNA and methods for reprogramming cells that exhibit a first differentiated state or phenotype to cells that exhibit a second differentiated state or phenotype, such as to reprogram human somatic cells to pluripotent stem cells.
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