NUCLEOBASE EDITORS AND USES THEREOF
    2.
    发明申请
    NUCLEOBASE EDITORS AND USES THEREOF 审中-公开
    核编辑器及其用途

    公开(公告)号:WO2017070632A3

    公开(公告)日:2017-06-08

    申请号:PCT/US2016058344

    申请日:2016-10-22

    Abstract: 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 single site 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 proteins or protein domains, e.g., deaminase domains, are provided. In some embodiments, methods for targeted nucleic acid editing 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 proteins or domains, are provided.

    Abstract translation: 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,提供了Cas9的融合蛋白和核酸编辑蛋白或蛋白结构域,例如脱氨酶结构域。 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的试剂和试剂盒,例如Cas9的融合蛋白和核酸编辑蛋白或结构域。

    PROTEASE-CLEAVAGE RESISTANT, SHIGA TOXIN A SUBUNIT EFFECTOR POLYPEPTIDES AND CELL-TARGETED MOLECULES COMPRISING THE SAME
    3.
    发明申请
    PROTEASE-CLEAVAGE RESISTANT, SHIGA TOXIN A SUBUNIT EFFECTOR POLYPEPTIDES AND CELL-TARGETED MOLECULES COMPRISING THE SAME 审中-公开
    抗氧化抗体,SHIGA毒素亚胺效应物多糖和包含其的细胞定向分子

    公开(公告)号:WO2015191764A1

    公开(公告)日:2015-12-17

    申请号:PCT/US2015/035179

    申请日:2015-06-10

    Abstract: The present invention provides protease-cleavage resistant molecules comprising Shiga toxin effector polypeptides capable of exhibiting potent, Shiga toxin functions ( e.g . subcellular routing and cytotoxicity). The present invention also provides protease-cleavage resistant, cell-targeted molecules for targeting specific cell types, e.g ., infected or malignant cells. Certain molecules of present invention are cytotoxic, and certain cell-targeted molecules of present invention may be used for the targeted killing of specific cell types and treatment of a variety of diseases, disorders, and conditions, including cancers, tumors, growth abnormalities, immune disorders, and microbial infections. Certain cell-targeted molecules of the invention exhibit improved, in vivo tolerability as compared to related cell-targeted molecules comprising protease-cleavage sensitive, wild-type, Shiga toxin effector polypeptides. The cell-targeted molecules of the invention can deliver additional materials, such as, e.g ., antigens, cytotoxic agents, and detection-promoting agents, into interiors of target cells.

    Abstract translation: 本发明提供蛋白酶切割抗性分子,其包括能显示有效的志贺毒素功能(例如亚细胞定位和细胞毒性)的志贺毒素效应子多肽。 本发明还提供用于靶向特定细胞类型(例如感染或恶性细胞)的蛋白酶切割抗性的细胞靶向分子。 本发明的某些分子是细胞毒性的,并且本发明的某些细胞靶向分子可用于靶向特异性细胞类型的杀伤和各种疾病,病症和病症的治疗,包括癌症,肿瘤,生长异常,免疫 病症和微生物感染。 与包含蛋白酶切割敏感的野生型志贺毒素效应物多肽的相关细胞靶向分子相比,本发明的某些细胞靶向分子表现出改进的体内耐受性。 本发明的细胞靶向分子可以向靶细胞的内部递送另外的材料,例如抗原,细胞毒性剂和检测促进剂。

    VECTORS HARBORING TOXIC GENES, METHODS AND USES THEREFOR
    6.
    发明申请
    VECTORS HARBORING TOXIC GENES, METHODS AND USES THEREFOR 审中-公开
    载体含有毒性基因,方法和用途

    公开(公告)号:WO2013085624A1

    公开(公告)日:2013-06-13

    申请号:PCT/US2012/060441

    申请日:2012-10-16

    Inventor: CHEN, Haifeng

    Abstract: The present disclosure describes nucleic acids, and viruses comprising such nucleic acids, for growing a toxic gene in an insect cell. These nucleic acids comprise a sequence encoding a toxic polypeptide, and an intron that interrupts the sequence, whereby the intron is spliced in mammalian cells but not in insect cells. Infection of mammalian cells but not insect cells with the nucleic acids or viruses can lead to expression of toxic levels of the toxic polypeptide in mammalian cells but not in insect cells. Viruses, such as an AAV or a baculovirus comprising a nucleic acid can be grown in insect cell lines in vitro and can be administered to a subject in need of therapy, such as a subject in need of cancer therapy.

    Abstract translation: 本公开描述了用于在昆虫细胞中生长毒性基因的核酸和包含此类核酸的病毒。 这些核酸包含编码毒性多肽的序列和中断该序列的内含子,由此内含子在哺乳动物细胞中剪接而不在昆虫细胞中剪接。 哺乳动物细胞而不是昆虫细胞与核酸或病毒的感染可导致在哺乳动物细胞中毒性水平的毒性水平的表达,但不能在昆虫细胞中表达。 病毒如AAV或包含核酸的杆状病毒可以在昆虫细胞系中体外生长,并且可以施用于需要治疗的受试者,例如需要癌症治疗的受试者。

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