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公开(公告)号：WO2023086938A2
公开(公告)日：2023-05-19
申请号：PCT/US2022/079715
申请日：2022-11-11
IPC分类号： C12N9/22 , C12N15/11 , C12N15/90 , A61K38/46 , A61K31/7088
摘要： The present invention relates to nucleases or nucleic acids encoding the nucleases, RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the nucleases and/or RNA guides, compositions comprising the nucleases and/or RNA guides, and kits and/or methods for preparing and/or using the nucleases and/or RNA guides.
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2.发明申请
公开(公告)号：WO2023018856A1
公开(公告)日：2023-02-16
申请号：PCT/US2022/040032
申请日：2022-08-11
发明人： DITOMMASO, Tia, Marie , HASWELL, Jeffrey, Raymond , JAKIMO, Noah, Michael , SENGUPTA, Sejuti , WESSELLS, Quinton, Norman
IPC分类号： C12N15/113
摘要： A system for genetic editing of a polypyrimidine tract binding protein 1 (PTBP1) gene, comprising (i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, and (ii) an RNA guide or a second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an PTBP1 gene. Also provided herein are methods for editing a PTBP1 gene using the gene editing system disclosed herein and/or for treating diseases associated with the PTBP1 gene.
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3.发明申请
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：WO2022159741A1
公开(公告)日：2022-07-28
申请号：PCT/US2022/013375
申请日：2022-01-21
IPC分类号： A61K38/46 , A61K48/00 , C07K19/00 , C12N15/113
摘要： The present invention relates to nucleases or nucleic acids encoding the nucleases, RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the nucleases and/or RNA guides, compositions comprising the nucleases and/or RNA guides, and kits and/or methods for preparing and/or using the nucleases and/or RNA guides.
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公开(公告)号：WO2022150608A1
公开(公告)日：2022-07-14
申请号：PCT/US2022/011647
申请日：2022-01-07
摘要： The present invention relates to variant CRISPR nuclease polypeptides, methods of preparing the variant CRISPR nuclease polypeptides, processes for characterizing the variant CRISPR nuclease polypeptides, compositions and cells comprising the variant CRISPR nuclease polypeptides, and methods of using the variant CRISPR nuclease polypeptides. The invention further relates to complexes comprising a variant CRISPR nuclease polypeptide, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
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公开(公告)号：WO2021041569A1
公开(公告)日：2021-03-04
申请号：PCT/US2020/048037
申请日：2020-08-26
IPC分类号： C07K14/195 , C07K19/00 , C12N9/22
摘要： The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturaliy occurring, engineered CRISPR systems, 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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公开(公告)号：WO2019090173A1
公开(公告)日：2019-05-09
申请号：PCT/US2018/059089
申请日：2018-11-02
IPC分类号： C12N9/22 , C12N15/10 , C12N15/90 , C12N15/113 , C07K14/195
摘要： The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of DNA, RNA, and protein substrates. Each system includes one or more protein components and one or more nucleic acid components that together target DNA, RNA, or protein substrates.
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公开(公告)号：WO2023086965A2
公开(公告)日：2023-05-19
申请号：PCT/US2022/079755
申请日：2022-11-11
IPC分类号： C12N9/22 , C12N15/113 , C12N15/10
摘要： The present invention relates to nucleases or nucleic acids encoding the nucleases, RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the nucleases and/or RNA guides, compositions comprising the nucleases and/or RNA guides, and kits and/or methods for preparing and/or using the nucleases and/or RNA guides.
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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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