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    Regulatory elements for delivery to the liver
    1.
    发明授权
    Regulatory elements for delivery to the liver 有权
    用于输送到肝脏的调节元件

    公开(公告)号：US07312324B2

    公开(公告)日：2007-12-25

    申请号：US10139763

    申请日：2002-05-06

    申请人： David W. Souza ,  Donna Armentano ,  Samuel C. Wadsworth

    发明人： David W. Souza ,  Donna Armentano ,  Samuel C. Wadsworth

    IPC分类号： C07H21/04 ,  C07H21/02 ,  A01N63/00 ,  A01N65/00 ,  A01N43/04 ,  A01N31/70

    CPC分类号： C12N15/86 ,  A61K48/00 ,  A61K48/0058 ,  C12N15/85 ,  C12N2710/10343 ,  C12N2799/022 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/85

    摘要： The invention is directed to novel combinations of liver specific enhancers and promoter elements for achieving persistent transgene expression in the liver. The liver specific enhancer elements may be derived from either the human serum albumin, prothrombin, α-1microglobulin or aldolase genes in single copies or in multimerized form linked to elements derived from the cytomegalovirus intermediate early (CMV), α-1-antitrypsin or albumin promoters. In a preferred embodiment of the invention, an adenoviral vector comprising a liver specific enhancer/promoter combination operably linked to a transgene is administered to recipient cells. In other embodiments of the invention, adeno-associated viral vectors, retroviral vectors, lentiviral vectors or a plasmid comprising the liver specific enhancer/promoter combination linked to a transgene is administered to recipient cells. Also within the scope of the invention are promoter elements derived from the human prothrombin gene and the β-fibrinogen gene.

    摘要翻译： 本发明涉及用于实现肝脏中持续转基因表达的肝特异性增强子和启动子元件的新型组合。 肝脏特异性增强子元件可以来源于与来自巨细胞病毒中间体早期（CMV），α-1-抗胰蛋白酶或白蛋白的元件连接的单拷贝或多聚化形式的人血清白蛋白，凝血酶原，α-1微球蛋白或醛缩酶基因 发起人。 在本发明的优选实施方案中，向受体细胞施用包含可操作地连接到转基因的肝特异性增强子/启动子组合的腺病毒载体。 在本发明的其它实施方案中，将腺相关病毒载体，逆转录病毒载体，慢病毒载体或包含与转基因连接的肝特异性增强子/启动子组合的质粒施用于受体细胞。 也在本发明范围内的是源自人凝血酶原基因和β-纤维蛋白原基因的启动子元件。

    AAV vectors for gene therapy
    2.
    发明授权
    AAV vectors for gene therapy 有权
    用于基因治疗的AAV载体

    公开(公告)号：US06632670B1

    公开(公告)日：2003-10-14

    申请号：US09029705

    申请日：1998-11-16

    申请人： Samuel C. Wadsworth ,  Karen Vincent ,  Susan Piraino ,  Sirkka Kyostio

    发明人： Samuel C. Wadsworth ,  Karen Vincent ,  Susan Piraino ,  Sirkka Kyostio

    IPC分类号： C12N15864

    CPC分类号： C12N7/00 ,  A01K2217/05 ,  A61K48/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14152

    摘要： The present invention is directed to methods for generating high titer, contaminant free, recombinant AAV vectors, methods and genetic constructs for producing recombinant AAV vectors conveniently and in large quantities, methods for the delivery of all essential viral proteins required in trans for high yields of recombinant AAV, recombinant AAV vectors for use in gene therapy, novel packaging cell lines which obviate the need for cotransfection of vector and helper plasmids, helper plasmids and vector plasmid backbone constructs, a reporter assay for determining AAV vector yield. Further provided are recombinant AAV vectors in a pharmaceutically acceptable carrier, methods of delivering a transgene of interest to a cell, compositions and methods for delivering a DNA sequence encoding a desired polypeptide to a cell, and transgenic non-human mammals that express a human chromosome 19 AAV integration locus.

    摘要翻译： 本发明涉及用于产生高效价，无污染的重组AAV载体的方法，用于方便和大量生产重组AAV载体的方法和遗传构建体，用于递送反式所需的所有必需病毒蛋白的高产率的方法 重组AAV，用于基因治疗的重组AAV载体，新型包装细胞系，其不需要共转染载体和辅助质粒，辅助质粒和载体质粒骨架构建体，用于测定AAV载体产量的报道分析。 还提供了药学上可接受的载体中的重组AAV载体，将感兴趣的转基因递送至细胞的方法，用于将编码所需多肽的DNA序列递送至细胞的组合物和方法，以及表达人染色体的转基因非人哺乳动物 19 AAV整合轨迹。

    Adenoviral helper vectors
    3.
    发明授权
    Adenoviral helper vectors 失效
    腺病毒辅助载体

    公开(公告)号：US06541245B1

    公开(公告)日：2003-04-01

    申请号：US09718034

    申请日：2000-11-21

    申请人： Helen Romanczuk ,  Samuel C. Wadsworth ,  Patricia Berthelette

    发明人： Helen Romanczuk ,  Samuel C. Wadsworth ,  Patricia Berthelette

    IPC分类号： C12N1573

    CPC分类号： C12N7/00 ,  C12N15/86 ,  C12N2710/10343 ,  C12N2710/10352 ,  C12N2800/108 ,  C12N2800/30 ,  C12N2830/38 ,  C12N2830/42

    摘要： The present invention is directed to improved helper vectors and cell lines for the production of pseudoadenoviral (PAV) vectors containing substantially reduced levels of contaminating helper vector. The invention provides for helper vectors for the production of substantially helper vector-free PAV stocks comprising phag C31 recombinase recognition sequences which, depending upon their arrangement within the helper vector, can prevent helper vector packaging. The invention also provides for improved cell lines for the production of substantially helper vector-free PAV stocks comprising a stably introduced novel circular PAV genome into the cell.

    摘要翻译： 本发明涉及用于生产含有显着降低的污染辅助载体水平的假性腺病毒（PAV）载体的改良的辅助载体和细胞系。 本发明提供了用于生产基本上无辅助载体的PAV原液的辅助载体，其包含phag C31重组酶识别序列，其依赖于其在辅助载体内的排列可以防止辅助载体包装。 本发明还提供用于生产基本上无辅助载体的PAV种群的改良细胞系，其包含稳定引入的新的环状PAV基因组进入细胞。

    Methods for treating blood coagulation disorders
    5.
    发明授权
    Methods for treating blood coagulation disorders 有权
    治疗凝血障碍的方法

    公开(公告)号：US07615537B2

    公开(公告)日：2009-11-10

    申请号：US10057620

    申请日：2001-10-25

    申请人： Abraham Scaria ,  Samuel C. Wadsworth

    发明人： Abraham Scaria ,  Samuel C. Wadsworth

    IPC分类号： A61K48/00 ,  C12N15/63

    CPC分类号： C12N9/6437 ,  A61K48/00 ,  C07K2319/02 ,  C12N2799/021 ,  C12Y304/21021

    摘要： The present invention relates to a method of treating an individual having a blood coagulation defect (e.g., hemophilia A, hemophilia B), comprising administering to the individual an effective amount of a DNA vector encoding modified Factor VII (FVII), wherein the modified Factor VII leads to generation of Factor VIIa in vivo. In a particular embodiment, the invention pertains to a method of treating an individual having a blood coagulation defect comprising administering to the individual an effective amount of a nucleic acid encoding a modified FVII wherein the modified FVII comprises a signal which codes for precursor cleavage by furin at the activation cleavage site of the modified FVII. The invention also relates to a method of treating an individual having a blood coagulation disorder comprising administering to the individual an effective amount of a nucleic acid encoding the light chain of human FVII and a nucleic acid encoding the heavy chain of human FVII operably linked to a leader sequence. Compositions, expression vectors and host cells comprising nucleic acid which encodes a modified Factor VII, wherein the modified Factor VII leads to generation of Factor VIIa in vivo is also encompassed by the present invention.

    摘要翻译： 本发明涉及一种治疗患有血液凝固缺陷的个体（例如，血友病A，血友病B）的方法，包括向个体施用有效量的编码修饰因子VII（FVII）的DNA载体，其中修饰因子 VII导致体内因子VIIa的产生。 在一个具体实施方案中，本发明涉及治疗具有凝血缺陷的个体的方法，其包括向个体施用有效量的编码修饰的FVII的核酸，其中修饰的FVII包含编码弗林蛋白酶前体切割的信号 在修饰的FVII的激活切割位点。 本发明还涉及一种治疗患有血液凝固病症的个体的方法，包括向个体施用有效量的编码人FVII轻链的核酸和编码人FVII重链的核酸，其可操作地连接到 领导序列。 包含编码修饰因子VII的核酸的组合物，表达载体和宿主细胞，其中修饰的因子VII导致体内产生因子VIIa也包括在本发明中。