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公开(公告)号:US20240002878A1
公开(公告)日:2024-01-04
申请号:US18327373
申请日:2023-06-01
IPC分类号: C12N15/85 , C12N9/22 , C12N15/11 , C12N15/115 , C12N15/62
CPC分类号: C12N15/85 , C12N9/22 , C12N15/11 , C12N15/115 , C12N15/62 , C12N2310/20 , C12N2800/80 , C12N15/907
摘要: Aspects of the disclosure relate to methods and synthetic regulatory systems for more efficient nuclease-mediated homology-directed repair (HDR). In particular, provided herein are methods for more efficient in vivo and in vitro HDR-based gene editing where the methods comprise introducing into a cell a synthetic regulatory system comprising Cas nuclease, guide RNAs (gRNAs) having various lengths and configured to target distinct nucleotide sequences for simultaneous transcriptional repression (or activation) and genome editing via double stranded break and use of a donor nucleic acid molecule as a template for repair.
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公开(公告)号:US12077771B2
公开(公告)日:2024-09-03
申请号:US18327373
申请日:2023-06-01
CPC分类号: C12N15/85 , C12N9/22 , C12N15/11 , C12N15/115 , C12N15/62 , C12N15/907 , C12N2310/128 , C12N2310/16 , C12N2310/20 , C12N2501/12 , C12N2501/405 , C12N2800/80
摘要: Aspects of the disclosure relate to methods and synthetic regulatory systems for more efficient nuclease-mediated homology-directed repair (HDR). In particular, provided herein are methods for more efficient in vivo and in vitro HDR-based gene editing where the methods comprise introducing into a cell a synthetic regulatory system comprising Cas nuclease, guide RNAs (gRNAs) having various lengths and configured to target distinct nucleotide sequences for simultaneous transcriptional repression (or activation) and genome editing via double stranded break and use of a donor nucleic acid molecule as a template for repair.
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