公开(公告)号：US20140363876A1

公开(公告)日：2014-12-11

申请号：US14471649

申请日：2014-08-28

Applicant: CELLSCRIPT, LLC

Inventor： Jerome Jendrisak ,  Ronald Meis ,  Gary Dahl

IPC: C12P19/34

CPC classification number: 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

Abstract: 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.

公开(公告)号：US20240181005A1

公开(公告)日：2024-06-06

申请号：US17998752

申请日：2021-05-17

Applicant: CellScript, LLC ,  THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVIC

Inventor： Ronald Meis ,  Gary Dahl ,  Suk See De Ravin ,  Julie Brault ,  Colin L. Sweeney ,  Harry L. Malech

IPC: 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 classification number: 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

Abstract: 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.

公开(公告)号：US09005930B2

公开(公告)日：2015-04-14

申请号：US14471649

申请日：2014-08-28

Applicant: Cellscript, LLC

Inventor： Jerome Jendrisak ,  Ronald Meis ,  Gary Dahl

IPC: C12P19/34 ,  C12P21/06

CPC classification number: 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

Abstract: 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.

公开(公告)号：US20140221248A1

公开(公告)日：2014-08-07

申请号：US14185384

申请日：2014-02-20

Applicant: CELLSCRIPT, LLC

Inventor： Jerome Jendrisak ,  Ronald Meis ,  Gary Dahl

IPC: C12N15/10

CPC classification number: 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

Abstract: 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.

Abstract translation: 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。

公开(公告)号：US09115380B2

公开(公告)日：2015-08-25

申请号：US14666805

申请日：2015-03-24

Applicant: CELLSCRIPT, LLC

Inventor： Jerome Jendrisak ,  Ronald Meis ,  Gary Dahl

IPC: C12P19/34 ,  C12P21/06 ,  C12N9/12 ,  C12N9/10

CPC classification number: 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

Abstract: 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.

Abstract translation: 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。

公开(公告)号：US20150191760A1

公开(公告)日：2015-07-09

申请号：US14666805

申请日：2015-03-24

Applicant: CELLSCRIPT, LLC

Inventor： Jerome Jendrisak ,  Ronald Meis ,  Gary Dahl

IPC: C12P19/34 ,  C12N9/10 ,  C12N9/12

CPC classification number: 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

Abstract: 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.

Abstract translation: 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。

公开(公告)号：US08846348B2

公开(公告)日：2014-09-30

申请号：US14185384

申请日：2014-02-20

Applicant: CellScript, LLC

Inventor： Jerome Jendrisak ,  Ronald Meis ,  Gary Dahl

IPC: C12P19/34 ,  C12P21/06 ,  C12N15/10

CPC classification number: 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

Abstract: 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.

Abstract translation: 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。