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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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公开(公告)号:US20220257795A1
公开(公告)日:2022-08-18
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 , C12P21/02 , A61K38/00 , A61K31/7105 , C12P19/34 , C12N5/074 , C12N15/10 , C12N5/0793 , A61K33/06 , 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:US20210079346A1
公开(公告)日:2021-03-18
申请号:US17034999
申请日:2020-09-28
申请人: CellScript, LLC
发明人: Cynthia Chin , Anthony D. Person , Gary A. Dahl
IPC分类号: C12N5/078 , A61K38/16 , C07K14/705 , C07K14/715 , C07K14/005 , A61K31/7105 , A61K38/17 , A61K38/21 , A61K31/711 , C12N5/077 , C12N5/074
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:US10793831B2
公开(公告)日:2020-10-06
申请号:US15823869
申请日:2017-11-28
申请人: CellScript, LLC
发明人: Cynthia Chin , Anthony D. Person , Gary A. Dahl
IPC分类号: C12N5/078 , A61K38/16 , C07K14/705 , C07K14/715 , C07K14/005 , A61K31/7105 , A61K38/17 , A61K38/21 , A61K31/711 , C12N5/077 , C12N5/074
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:US10201620B2
公开(公告)日:2019-02-12
申请号:US14368399
申请日:2012-12-31
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 , C12N15/87 , C12P19/34 , C12N5/074 , C12N15/10 , C12N5/0793 , A61K33/06 , C12P21/02 , C12N5/077 , C12P19/38 , A61K38/00
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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公开(公告)号:US09115380B2
公开(公告)日:2015-08-25
申请号:US14666805
申请日:2015-03-24
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.
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:US20150191760A1
公开(公告)日:2015-07-09
申请号:US14666805
申请日:2015-03-24
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.
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:US08846348B2
公开(公告)日:2014-09-30
申请号:US14185384
申请日:2014-02-20
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.
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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