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    Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies
    4.
    发明授权
    Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies 有权
    编码人球蛋白基因的载体及其在治疗血红蛋白病中的应用

    公开(公告)号：US08058061B2

    公开(公告)日：2011-11-15

    申请号：US12433412

    申请日：2009-04-30

    申请人： Michel Sadelain ,  Stefano Rivella ,  Chad May ,  Joseph Bertino

    发明人： Michel Sadelain ,  Stefano Rivella ,  Chad May ,  Joseph Bertino

    IPC分类号： C12N5/00 ,  C12N15/00 ,  C12N15/67 ,  C12N15/11

    CPC分类号： C12N15/86 ,  A61K48/00 ,  A61K48/0066 ,  A61K2035/124 ,  C07K14/805 ,  C12N2510/00 ,  C12N2740/15043

    摘要： Recombinant lentiviral vectors having a region encoding a functional β-globin gene; and large portions of the β-globin locus control regions which include DNase I hypersensitive sites HS2, HS3 and HS4 provides expression of β-globin when introduced into a mammal, for example a human, in vivo. Optionally, the vector further includes a region encoding a dihydrofolate reductase. The vector may be used in treatment of hemoglobinopathies, including β-thalessemia and sickle-cell disease. For example, hematopoietic progenitor or stem cells may be transformed ex vivo and then restored to the patient. Selection processes may be used to increase the percentage of transformed cells in the returned population. For example, a selection marker which makes transformed cells more drug resistant than untransformed cells allows selection by treatment of the cells with the corresponding drug.

    摘要翻译： 具有编码功能性和珠蛋白基因的区域的重组慢病毒载体; 并且包括DNase I超敏感位点HS2，HS3和HS4的大部分的 - 珠蛋白基因座控制区在体内被引入到哺乳动物例如人中时提供了β-珠蛋白的表达。 任选地，载体还包括编码二氢叶酸还原酶的区域。 该载体可用于治疗血红蛋白病，包括疟原虫和镰状细胞病。 例如，造血祖细胞或干细胞可以离体转化，然后恢复到患者体内。 选择过程可用于增加返回群体中转化细胞的百分比。 例如，使转化细胞比未转化细胞更耐药的选择标记允许通过用相应的药物处理细胞进行选择。

    In-vivo energy depleting strategies for killing drug-resistant cancer cells
    5.
    发明申请
    In-vivo energy depleting strategies for killing drug-resistant cancer cells 失效
    用于杀死耐药性癌细胞的体内能量消耗策略

    公开(公告)号：US20050159387A1

    公开(公告)日：2005-07-21

    申请号：US10518003

    申请日：2003-06-13

    申请人： Daniel Martin ,  Joseph Bertino ,  Jason Koutcher

    发明人： Daniel Martin ,  Joseph Bertino ,  Jason Koutcher

    IPC分类号： A61K31/19 ,  A61K31/455 ,  A61K31/51 ,  A61K31/57 ,  A61K31/573 ,  A61K31/66 ,  A61K31/7072 ,  A61K31/7076 ,  A61K38/18 ,  A61K45/06

    CPC分类号： A61K45/06 ,  A61K31/455 ,  A61K31/57 ,  A61K31/7072 ,  A61K31/7076 ,  A61K38/193 ,  A61K2300/00

    摘要： This invention also provides a method for treating a cancer subject comprising administering to the subject a combination of ATP-depleting agents at concentrations which deplete the ATP level to, or close to, at least 15% of normal in cancer cells wherein at least one of the ATP-depleting agents is a mitochondrial ATP-inhibitor, a methylthioadenosine phosphorylase inhibitor or an inhibitor of De Novo purine synthesis other than 6-Methylmercaptopurine riboside, wherein said composition produces a substantially better effect than a composition without at least one of the ATP-depleting agents: a mitochondrial ATP-inhibitor, a glycolytic inhibitor, a methylthioadenosine phosphorylase inhibitor and an inhibitor of De Novo purine synthesis other than 6-Methylmercaptopurine riboside.

    摘要翻译： 本发明还提供了一种治疗癌症受试者的方法，其包括将ATP水平降低至或接近癌细胞中至少15％正常的浓度的ATP消耗剂的组合给予受试者，其中至少一种 ATP消耗剂是线粒体ATP抑制剂，甲基硫代腺苷磷酸化酶抑制剂或除6-甲基巯基嘌呤核苷之外的新诺夫嘌呤合成抑制剂，其中所述组合物产生比不含ATP- 消耗剂：线粒体ATP抑制剂，糖酵解抑制剂，甲基硫代腺苷磷酸化酶抑制剂和除6-甲基巯基嘌呤核苷之外的德诺嘌呤合成抑制剂。

    VECTOR ENCODING HUMAN GLOBIN GENE AND USE THEREOF IN TREATMENT OF HEMOGLOBINOPATHIES
    6.
    发明申请
    VECTOR ENCODING HUMAN GLOBIN GENE AND USE THEREOF IN TREATMENT OF HEMOGLOBINOPATHIES 有权
    编码人GLOBIN基因的载体及其在治疗肝细胞病变中的应用

    公开(公告)号：US20090274671A1

    公开(公告)日：2009-11-05

    申请号：US12433412

    申请日：2009-04-30

    申请人： Michel Sadelain ,  Stefano Rivella ,  Chad May ,  Joseph Bertino

    发明人： Michel Sadelain ,  Stefano Rivella ,  Chad May ,  Joseph Bertino

    IPC分类号： A61K35/12 ,  A61K31/7088 ,  C12N5/06 ,  C12N5/08 ,  C12N15/87

    CPC分类号： C12N15/86 ,  A61K48/00 ,  A61K48/0066 ,  A61K2035/124 ,  C07K14/805 ,  C12N2510/00 ,  C12N2740/15043

    摘要： Recombinant lentiviral vectors having a region encoding a functional β-globin gene; and large portions of the β-globin locus control regions which include DNase I hypersensitive sites HS2, HS3 and HS4 provides expression of β-globin when introduced into a mammal, for example a human, in vivo. Optionally, the vector further includes a region encoding a dihydrofolate reductase. The vector may be used in treatment of hemoglobinopathies, including β-thalessemia and sickle-cell disease. For example, hematopoietic progenitor or stem cells may be transformed ex vivo and then restored to the patient. Selection processes may be used to increase the percentage of transformed cells in the returned population. For example, a selection marker which makes transformed cells more drug resistant than untransformed cells allows selection by treatment of the cells with the corresponding drug.

    摘要翻译： 具有编码功能性β-珠蛋白基因的区域的重组慢病毒载体; 并且包括DNA酶I超敏感位点HS2，HS3和HS4的大部分β-珠蛋白基因座控制区在体内引入哺乳动物例如人体时提供β-珠蛋白的表达。 任选地，载体还包括编码二氢叶酸还原酶的区域。 载体可用于治疗血红蛋白病，包括β-贫血和镰状细胞病。 例如，造血祖细胞或干细胞可以离体转化，然后恢复到患者体内。 选择过程可用于增加返回群体中转化细胞的百分比。 例如，使转化细胞比未转化细胞更耐药的选择标记允许通过用相应的药物处理细胞进行选择。

    Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies
    7.
    发明授权
    Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies 有权
    编码人球蛋白基因的载体及其在治疗血红蛋白病中的应用

    公开(公告)号：US07541179B2

    公开(公告)日：2009-06-02

    申请号：US10188221

    申请日：2002-07-01

    申请人： Michel Sadelain ,  Stefano Rivella ,  Chad May ,  Joseph Bertino

    发明人： Michel Sadelain ,  Stefano Rivella ,  Chad May ,  Joseph Bertino

    IPC分类号： C12N15/00 ,  C12N15/10 ,  C12N15/67 ,  C12N7/01

    CPC分类号： C12N15/86 ,  A61K48/00 ,  A61K48/0066 ,  A61K2035/124 ,  C07K14/805 ,  C12N2510/00 ,  C12N2740/15043

    摘要： Recombinant lentiviral vectors having a region encoding a functional β-globin gene; and large portions of the β-globin locus control regions which include DNase I hypersensitive sites HS2, HS3 and HS4 provides expression of β-globin when introduced into a mammal, for example a human, in vivo. Optionally, the vector further includes a region encoding a dihydrofolate reductase. The vector may be used in treatment of hemoglobinopathies, including β-thalessemia and sickle-cell disease. For example, hematopoietic progenitor or stem cells may be transformed ex vivo and then restored to the patient. Selection processes may be used to increase the percentage of transformed cells in the returned population. For example, a selection marker which makes transformed cells more drug resistant than un-transformed cells allows selection by treatment of the cells with the corresponding drug.

    摘要翻译： 具有编码功能性β-珠蛋白基因的区域的重组慢病毒载体; 并且包括DNA酶I超敏感位点HS2，HS3和HS4的大部分β-珠蛋白基因座控制区在体内引入哺乳动物例如人体时提供β-珠蛋白的表达。 任选地，载体还包括编码二氢叶酸还原酶的区域。 载体可用于治疗血红蛋白病，包括β-贫血和镰状细胞病。 例如，造血祖细胞或干细胞可以离体转化，然后恢复到患者体内。 选择过程可用于增加返回群体中转化细胞的百分比。 例如，使转化细胞比未转化细胞更耐药的选择标记允许通过用相应的药物处理细胞进行选择。

    Method for modulating the production of a selected protein in vivo
    8.
    发明申请
    Method for modulating the production of a selected protein in vivo 审中-公开
    调节体内所选蛋白质生成的方法

    公开(公告)号：US20060003936A1

    公开(公告)日：2006-01-05

    申请号：US11213368

    申请日：2005-08-26

    申请人： Philipp Mayer-Kuckuk ,  Debabrata Banerjee ,  Joseph Bertino

    发明人： Philipp Mayer-Kuckuk ,  Debabrata Banerjee ,  Joseph Bertino

    IPC分类号： C07H21/04 ,  C12N9/12 ,  C12P21/06 ,  C07K14/705

    CPC分类号： C12N9/003 ,  C07K2319/00

    摘要： A method is provided for use in producing a selected protein in mammalian cells, and to cDNA molecules useful in the method, and fusion proteins produced from expression of the cDNA. In the method, cDNA encoding a fusion protein that includes a mammalian DHFR and the selected protein is introduced into mammalian cells such that it is expressed. The naturally occurring repression of DHFR translation is overcome by treatment of the cells with a folate or antifolate or similar composition. The relief from this repression extends to the selected protein which is the second part of the expressed fusion, such that the treatment results in controllable and enhanced production of the selected protein.

    摘要翻译： 提供了用于在哺乳动物细胞中产生所选蛋白质的方法，以及用于该方法的cDNA分子以及由cDNA表达产生的融合蛋白质。 在该方法中，将包含哺乳动物DHFR和所选蛋白质的融合蛋白的cDNA导入哺乳动物细胞，使其表达。 通过用叶酸或抗叶酸或类似组合物处理细胞来克服天然存在的DHFR翻译的抑制。 这种抑制的缓解延伸到所选择的蛋白质，其是表达的融合物的第二部分，使得治疗导致所选蛋白质的可控和增强的产生。