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公开(公告)号:WO2023081377A2
公开(公告)日:2023-05-11
申请号:PCT/US2022/048989
申请日:2022-11-04
发明人: WESSELLS, Quinton Norman , DITOMMASO, Tia Marie , HASWELL, Jeffrey Raymond , JAKIMO, Noah Michael
摘要: The present invention relates to compositions comprising RNA guides targeting CIITA, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.
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公开(公告)号:WO2022256642A2
公开(公告)日:2022-12-08
申请号:PCT/US2022/032144
申请日:2022-06-03
发明人: WESSELLS, Quinton Norman , HASWELL, Jeffrey Raymond , DITOMMASO, Tia Marie , JAKIMO, Noah Michael , SENGUPTA, Sejuti
IPC分类号: C12N15/113 , A61K48/00 , C12N9/22 , A61K38/46 , C12N15/1137 , C12N2310/20 , C12N2320/34 , C12Y101/03015
摘要: Provided herein are gene editing systems and/or compositions comprising RNA guides targeting HAO1 for use in genetic editing of the HAO1 gene. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
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公开(公告)号:WO2022162623A1
公开(公告)日:2022-08-04
申请号:PCT/IB2022/050783
申请日:2022-01-28
IPC分类号: C07K14/195 , C07K19/00 , C12N15/10 , C12N15/11 , C12N15/113
摘要: The present disclosure relates to systems, compositions and methods for modifying target nucleic acid sequences.
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公开(公告)号:WO2023039534A2
公开(公告)日:2023-03-16
申请号:PCT/US2022/076216
申请日:2022-09-09
摘要: The present invention relates to compositions comprising a Cas12i polypeptide, a deaminase polypeptide, and an RNA guide, processes for characterizing the compositions, cells comprising the compositions, Cas12i fusion proteins, Cas12i complexes, and methods of using the compositions.
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公开(公告)号:WO2023039472A2
公开(公告)日:2023-03-16
申请号:PCT/US2022/076112
申请日:2022-09-08
发明人: CHONG, Shaorong , WESSELLS, Quinton Norman , ZIBLAT, Roy , JAKIMO, Noah Michael , GARRITY, Anthony James , ALFONSE, Lauren E.
摘要: The present disclosure relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The disclosure further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
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6.
公开(公告)号:WO2022256655A2
公开(公告)日:2022-12-08
申请号:PCT/US2022/032162
申请日:2022-06-03
发明人: WESSELLS, Quinton Norman , HASWELL, Jeffrey Raymond , DITOMMASO, Tia Marie , JAKIMO, Noah Michael , SENGUPTA, Sejuti
IPC分类号: C12N15/113 , C12N15/90 , C12N9/22 , A61K48/00 , A61K38/46 , C12N15/1137 , C12N2310/20 , C12N2320/34 , C12Y101/01027
摘要: Provided herein are gene editing systems and/or compositions comprising RNA guides targeting LDHA for use in genetic editing of the LDHA gene. Also provide herein are methods of using the gene editing system for introducing edits to the LDHA gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
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公开(公告)号:WO2022162622A1
公开(公告)日:2022-08-04
申请号:PCT/IB2022/050782
申请日:2022-01-28
IPC分类号: C12N9/22 , C12N15/90 , C12N15/11 , C12N15/113 , C12N9/00
摘要: The present disclosure relates to systems, compositions and methods for modifying target nucleic acid sequences.
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公开(公告)号:WO2022159585A1
公开(公告)日:2022-07-28
申请号:PCT/US2022/013133
申请日:2022-01-20
发明人: HILBERT, Brendan Jay , JAKIMO, Noah Michael , SCOTT, David A. , MCGAW, Colin Alexander , CARTE, Jason Michael
摘要: The disclosure describes Casl2i2 fusion proteins, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered Casl2i2 fusion proteins, components, and methods for targeted modification of nucleic acids. Each system, includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
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