CAS VARIANTS FOR GENE EDITING
    10.
    发明公开
    CAS VARIANTS FOR GENE EDITING 审中-公开
    CAS变体用于基因编辑

    公开(公告)号:EP3080265A1

    公开(公告)日:2016-10-19

    申请号:EP14825518.5

    申请日:2014-12-12

    IPC分类号: C12N9/78

    摘要: Some aspects of this disclosure provide strategies, systems, reagents, methods, and kits that are useful for the targeted editing of nucleic acids, including editing a nucleic acid encoding a mutant von Willebrand Factor protein to correct a point mutation associated with a disease or disorder, e.g., with von Willebrand disease. The methods provided are useful for correcting a vWF point mutation within the genome of a cell or subject, e.g., within the human genome. In some embodiments, fusion proteins of Cas9 and nucleic acid editing enzymes or enzyme domains, e.g., deaminase domains, are provided. In some embodiments, reagents and kits for the generation of targeted nucleic acid editing proteins, e.g., fusion proteins of Cas9 and nucleic acid editing enzymes or domains, are provided.

    摘要翻译: 本公开的一些方面提供可用于核酸的定向编辑的策略,系统,试剂,方法和试剂盒,包括编辑例如人基因组内的细胞或受试者基因组内的单个位点。 在一些实施方案中,Cas9的融合蛋白与核酸编辑酶或酶结构域例如 ,脱氨酶域,提供。 在一些实施方案中,提供了用于靶向核酸编辑的方法。 在一些实施方案中,提供用于产生靶向核酸编辑蛋白质的试剂和试剂盒,例如Cas9的融合蛋白和核酸编辑酶或结构域。