METHODS FOR ENGINEERING NON-NEURONAL CELLS INTO NEURONS AND USING NEWLY ENGINEERED NEURONS TO TREAT NEURODEGENERATIVE DISEASES

    公开(公告)号:US20190119673A1

    公开(公告)日:2019-04-25

    申请号:US16030022

    申请日:2018-07-09

    摘要: The invention provides compositions and in vivo, ex vivo and in vitro methods for trans-differentiation of or re-programming mammalian cells to functional neurons. In particular, the invention provides methods for engineering non-neuronal cells into neurons, including fully functional human neuronal cells, and methods for engineering non-neuronal cells into neurons, e.g., fully functional human neuronal cells, in the brain to treat a neurodegenerative disease. In alternative embodiments, the invention provides compositions comprising re-differentiated or re-programmed mammalian cells, such as human cells, of the invention. The invention also provides compositions and methods for direct reprogramming of cells to a second phenotype or differentiated phenotype, such as a neuron, including a fully functional human neuronal cell. The invention also provides formulations, products of manufacture, implants, artificial organs or tissues, or kits, comprising a trans-differentiated or re-programmed cell of the invention, e.g., a fully functional human neuronal cell.

    METHODS FOR ENGINEERING NON-NEURONAL CELLS INTO NEURONS AND USING NEWLY ENGINEERED NEURONS TO TREAT NEURODEGENERATIVE DISEASES
    6.
    发明申请
    METHODS FOR ENGINEERING NON-NEURONAL CELLS INTO NEURONS AND USING NEWLY ENGINEERED NEURONS TO TREAT NEURODEGENERATIVE DISEASES 审中-公开
    将非神经细胞工程化成神经元和使用新工程神经元治疗神经损伤性疾病的方法

    公开(公告)号:US20150299698A1

    公开(公告)日:2015-10-22

    申请号:US14439125

    申请日:2013-11-01

    摘要: The invention provides compositions and in vivo, ex vivo and in vitro methods for trans-differentiation of or re-programming mammalian cells to functional neurons. In particular, the invention provides methods for engineering non-neuronal cells into neurons, including fully functional human neuronal cells, and methods for engineering non-neuronal cells into neurons, e.g., fully functional human neuronal cells, in the brain to treat a neurodegenerative disease. In alternative embodiments, the invention provides compositions comprising re-differentiated or re-programmed mammalian cells, such as human cells, of the invention. The invention also provides compositions and methods for direct reprogramming of cells to a second phenotype or differentiated phenotype, such as a neuron, including a fully functional human neuronal cell. The invention also provides formulations, products of manufacture, implants, artificial organs or tissues, or kits, comprising a trans-differentiated or re-programmed cell of the invention, e.g., a fully functional human neuronal cell.

    摘要翻译: 本发明提供了用于将哺乳动物细胞反向分化或重新编程为功能性神经元的组合物和体内,离体和体外方法。 特别地,本发明提供了将非神经元细胞植入神经元,包括完全功能的人神经元细胞的方法,以及用于将非神经元细胞工程化成神经元的方法,例如在脑中完全功能的人神经元细胞以治疗神经变性疾病 。 在替代实施方案中,本发明提供包含本发明的再分化或重编程哺乳动物细胞(例如人细胞)的组合物。 本发明还提供用于将细胞直接重编程为第二表型或分化表型的组合物和方法,所述第二表型或分化表型包括神经元,包括完全功能的人神经元细胞。 本发明还提供了制剂,制剂产品,植入物,人造器官或组织或试剂盒,其包含本发明的跨分化或重编程细胞,例如完全功能的人神经元细胞。