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公开(公告)号：WO2022261149A2
公开(公告)日：2022-12-15
申请号：PCT/US2022/032578
申请日：2022-06-07
发明人： HIGGINS, Sean , COLIN, Isabel , SPINNER, Hannah , GARDNER, Matthew , GOMBERG, Trent , VIJAYAKUMAR, Gayathri , DENNY, Sarah , STAAHL, Brett T. , ADIL, Maroof , OAKES, Benjamin , SIDORE, Angus , MAKHIJA, Suraj
IPC分类号： A61K47/69 , C07K14/16 , C12N15/11 , C12N9/22 , C12N9/50 , C12N7/02 , C07K14/005 , C12N15/111 , C12N2310/16 , C12N2310/20 , C12N2310/3519 , C12N2320/32 , C12N2740/11022 , C12N2740/12022 , C12N2740/13022 , C12N2740/14022 , C12N2740/15023 , C12N2740/16022 , C12N2740/16043 , C12N2740/16222 , C12N2760/20122 , C12N2760/20222 , C12N2795/18122 , C12N7/00
摘要： Provided herein are delivery particle systems (XDP) useful for the delivery of payloads of any type. In some embodiments, a XDP particle system with tropism for target cells of interest is used to deliver CRISPR/Cas polypeptides (e.g., CasX proteins) and guide nucleic acids (gNA), for the modification of nucleic acids in target cells. Also provided are methods of making and using such XDP to modify the nucleic acids in such cells.
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公开(公告)号：WO2022261150A2
公开(公告)日：2022-12-15
申请号：PCT/US2022/032579
申请日：2022-06-07
发明人： HIGGINS, Sean , COLIN, Isabel , SPINNER, Hannah , GARDNER, Matthew , GOMBERG, Trent , VIJAYAKUMAR, Gayathri , DENNY, Sarah , STAAHL, Brett T. , ADIL, Maroof , OAKES, Benjamin , SIDORE, Angus , MAKHIJA, Suraj
IPC分类号： A61K47/69 , C07K14/16 , C12N15/11 , C12N9/22 , C12N9/50 , C12N7/02 , C07K14/005 , C12N15/111 , C12N2310/16 , C12N2310/20 , C12N2310/3519 , C12N2320/32 , C12N2740/11022 , C12N2740/12022 , C12N2740/13022 , C12N2740/14022 , C12N2740/15023 , C12N2740/16022 , C12N2740/16043 , C12N2740/16222 , C12N2760/20122 , C12N2760/20222 , C12N2795/18122 , C12N7/00
摘要： Provided herein are delivery particle systems (XDP) useful for the delivery of payloads of any type. In some embodiments, a XDP particle system with tropism for target cells of interest is used to deliver CRISPR/Cas polypeptides (e.g., CasX proteins) and guide nucleic acids (gNA), for the modification of nucleic acids in target cells. Also provided are methods of making and using such XDP to modify the nucleic acids in such cells.
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公开(公告)号：WO2023049872A2
公开(公告)日：2023-03-30
申请号：PCT/US2022/076980
申请日：2022-09-23
发明人： BANEY, Katherine , COLIN, Isabel , FORTUNY, Cecile , HIGGINS, Sean , MAKHIJA, Suraj , STAAHL, Brett T. , ADIL, Maroof , OAKES, Benjamin , WRIGHT, Addison , SIDORE, Angus , MOHR, Manuel , DENNY, Sarah
摘要： Provided herein are compositions and methods for use of self-inactivating recombinant vectors (SIRV) encoding Class 2 Type V and guide ribonucleic acid (gRNA) sequences useful for nucleic acid sequence editing, and including self-inactivating components. The SIRV may be delivered to cells as part of an AAV vector to target a gene of interest.
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公开(公告)号：WO2022125843A1
公开(公告)日：2022-06-16
申请号：PCT/US2021/062714
申请日：2021-12-09
发明人： MOHR, Manuel , BANEY, Katherine , SIDORE, Angus , FORTUNY, Cecile , ADIL, Maroof , WRIGHT, Addison , STAAHL, Brett T. , HIGGINS, Sean , OAKES, Benjamin , MAKHIJA, Suraj , DENNY, Sarah
IPC分类号： C12N9/22 , C12N15/63 , C12N15/90 , C12N15/864
摘要： Provided herein polynucleotides configured for incorporation into recombinant adeno-associated virus (AAV) vectors. The polynucleotides encode for CRISPR proteins, gRNA, and ancillary components of AAV vectors useful in the modification of target nucleic acids. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the target nucleic acid of a gene. Also provided are methods of using such AAV vectors to modify cells having such mutations.
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公开(公告)号：WO2022120095A1
公开(公告)日：2022-06-09
申请号：PCT/US2021/061673
申请日：2021-12-02
发明人： HIGGINS, Sean , COLIN, Isabel , DENNY, Sarah , STAAHL, Brett T. , OAKES, Benjamin , SIDORE, Angus , MAKHIJA, Suraj
IPC分类号： C12N15/113 , C12N9/22
摘要： Provided herein are engineered Class 2, Type V nucleases and guide RNAs useful for the editing of target nucleic acids. Also provided are methods of making and using such variants to modify nucleic acids.
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