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    MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
    2.
    发明申请
    MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES 审中-公开
    材料和方法治疗血红蛋白病

    公开(公告)号：WO2016135557A2

    公开(公告)日：2016-09-01

    申请号：PCT/IB2016/000276

    申请日：2016-02-23

    申请人： CRISPR THERAPEUTICS AG

    发明人： PORTEUS, Matthew, Hebden

    IPC分类号： C12N15/113 ,  C12N15/09 ,  C12N15/63

    CPC分类号： C12N15/113 ,  A61K35/28 ,  A61K35/545 ,  A61K38/42 ,  C12N5/0647 ,  C12N5/0696 ,  C12N15/102 ,  C12N15/63 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/11 ,  C12N2501/73 ,  C12N2510/00

    摘要： The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34 + human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.

    摘要翻译： 本申请提供了用于治疗血红蛋白病的材料和方法。 更具体地，本申请提供了产生祖细胞的方法，所述祖细胞通过基因组编辑来遗传修饰以增加胎儿血红蛋白（HbF）的产生，以及修饰的祖细胞（包括例如CD34 + 人造血干细胞）产生更高水平的HbF，以及使用这些细胞治疗血红蛋白病例如镰状细胞贫血症和β-地中海贫血的方法。

    METHOD OF PRODUCING LEUKOCYTES USING PTPN2 INHIBITION FOR ADOPTIVE CELL TRANSFER
    3.
    发明申请
    METHOD OF PRODUCING LEUKOCYTES USING PTPN2 INHIBITION FOR ADOPTIVE CELL TRANSFER 审中-公开
    使用PTPN2抑制自由基细胞转移制备白蛋白的方法

    公开(公告)号：WO2015188228A1

    公开(公告)日：2015-12-17

    申请号：PCT/AU2015/050318

    申请日：2015-06-10

    申请人： MONASH UNIVERSITY

    发明人： TIGANIS, Tony ,  WIEDE, Florian

    IPC分类号： A61K35/17 ,  C12N15/113 ,  C12N5/0783

    CPC分类号： C12N5/0638 ,  A61K35/17 ,  A61K39/0011 ,  A61K2039/5158 ,  A61K2039/572 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2501/51 ,  C12N2501/515 ,  C12N2501/73 ,  C12Y301/03048

    摘要： The present invention generally relates to methods of preparing leukocytes, particularly T cells, ex vivo for use in immunotherapy, particularly cancer immunotherapy. More specifically, the invention relates to methods for the preparation of leukocytes exhibiting cytotoxic properties for use in adoptive cell transfer. The invention also relates to cells and compositions including them for cancer immunotherapy. The invention also relates to methods of immunotherapy, particularly cancer immunotherapy. The present invention relates to a method for producing a leukocyte that has an enhanced capacity for killing a target cell, the method including contacting the leukocyte with a PTPN2 inhibitor in conditions for enabling the inhibitor to inactivate PTPN2 in the leukocyte, thereby producing a leukocyte that has an enhanced capacity for killing a target cell. Preferably, the leukocyte is contacted with the PTPN2 inhibitor in the absence of a T helper cell.

    摘要翻译： 本发明一般涉及制备用于免疫治疗，特别是癌症免疫疗法的白细胞，特别是T细胞的方法。 更具体地，本发明涉及用于制备用于过继细胞转移的细胞毒性的白细胞的制备方法。 本发明还涉及包含它们用于癌症免疫治疗的细胞和组合物。 本发明还涉及免疫治疗的方法，特别是癌症免疫治疗。 本发明涉及一种具有增强靶细胞杀伤能力的白细胞的制造方法，所述方法包括使白细胞与PTPN2抑制剂接触以使抑制剂能够使白细胞中的PTPN2失活的条件，从而产生白细胞， 具有增强的杀死靶细胞的能力。 优选地，在没有T辅助细胞的情况下，白细胞与PTPN2抑制剂接触。

    METABOLIC DOWNREGULATION FOR CELL SURVIVAL
    6.
    发明申请
    METABOLIC DOWNREGULATION FOR CELL SURVIVAL 审中-公开
    细胞生存的代谢下调

    公开(公告)号：WO2013134209A1

    公开(公告)日：2013-09-12

    申请号：PCT/US2013/029025

    申请日：2013-03-05

    申请人： WAKE FOREST UNIVERSITY HEALTH SCIENCES

    发明人： YOO, James ,  LEE, Sang, Jin ,  KIM, Jachyun ,  ATALA, Anthony

    IPC分类号： A61K31/70 ,  A01N1/02

    CPC分类号： A61K31/7076 ,  C12N5/0018 ,  C12N2500/40 ,  C12N2501/73

    摘要： Provided is a system and method of maintaining and/or increasing cell viability by downregulating cellular metabolic rate under hypoxic conditions via increasing and/or prolonging the availability of adenosine or derivatives thereof in the cell. Also provided is a system and method of prolonging the survival of implanted cells that are under hypoxic condition until host neovascularization is achieved, via increasing and/or prolonging the availability of adenosine or derivatives there in the cells. Also provided is a system and method of maintaining and/or increasing cell viability by downregulating cellular metabolic rate under hypoxic conditions via applying at least one purine metabolism enzyme inhibitor to the cell.

    摘要翻译： 本发明提供通过增加和/或延长细胞中腺苷或其衍生物的可用性，在低氧条件下下调细胞代谢速率来维持和/或增加细胞活力的系统和方法。 还提供了一种系统和方法，其通过增加和/或延长细胞中腺苷或衍生物的可用性来延长处于缺氧条件下的植入细胞的存活直到宿主新生血管形成。 还提供了通过向细胞施加至少一种嘌呤代谢酶抑制剂，通过在低氧条件下下调细胞代谢速率来维持和/或增加细胞活力的系统和方法。

    MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
    10.
    发明申请
    MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES 审中-公开
    材料和方法治疗血红蛋白病

    公开(公告)号：WO2016135557A3

    公开(公告)日：2016-10-20

    申请号：PCT/IB2016000276

    申请日：2016-02-23

    申请人： CRISPR THERAPEUTICS AG

    发明人： PORTEUS MATTHEW HEBDEN

    IPC分类号： C12N15/113 ,  C12N15/09 ,  C12N15/63

    CPC分类号： A61K38/42 ,  A61K35/28 ,  A61K35/545 ,  C12N5/0647 ,  C12N5/0696 ,  C12N15/102 ,  C12N15/113 ,  C12N15/63 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/11 ,  C12N2501/73 ,  C12N2510/00

    摘要： The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.

    摘要翻译： 本申请提供了用于治疗血红蛋白病的材料和方法。 更具体地说，本申请提供了产生通过基因组编辑增加胎儿血红蛋白（HbF）以及修饰的祖细胞（包括例如CD34 +人造血干细胞）的产生的祖细胞的方法，所述修饰的祖细胞产生增加的水平 的HbF，以及使用这些细胞治疗血红蛋白病例如镰状细胞贫血和β-地中海贫血的方法。