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    AAV vectors for gene therapy
    1.
    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。

    Silent Fc variants of anti-CD40 antibodies
    5.
    发明授权
    Silent Fc variants of anti-CD40 antibodies 有权
    抗CD40抗体的沉默Fc变体

    公开(公告)号：US09221913B2

    公开(公告)日：2015-12-29

    申请号：US14452647

    申请日：2014-08-06

    申请人： Christoph Heusser ,  James Rush ,  Karen Vincent

    发明人： Christoph Heusser ,  James Rush ,  Karen Vincent

    IPC分类号： A61K39/395 ,  C07K16/28 ,  A61K39/00

    CPC分类号： C07K16/2878 ,  A61K2039/505 ,  C07K16/2866 ,  C07K2317/52 ,  C07K2317/565 ,  C07K2317/60 ,  C07K2317/71

    摘要： Anti-CD40 antibodies have not been reported to induce hemostatic events in patients, however elevations in pancreatic enzymes in B cell lymphoma patients receiving the anti-CD40 Ab Chir12.12 and the possible risk of pancreatitis precludes the use of this Fc-competent anti-CD40 antibody in chronic autoimmune disease and transplantation for safety reasons. We therefore generated Fc-silent IgG1 anti-CD40 antibodies (mAb1, mAb2 and mAb3) unable to mediate antibody-dependent cellular cytotoxicity (ADCC) or complement-dependent cytotoxicity (CDC) both in vitro and in vivo. mAb1 was able to prolong non-human primate renal allograft survival in combination with sub-therapeutic doses of cyclosporine. In addition, mAb1 was able to completely suppress primary and secondary antibody responses to immunization with a T cell-dependent antigen. Crucially, there was no evidence of hemostatic events or abnormal pancreatic histology in either the transplant or immunization study. Collectively these results suggest mAb1 would be a safe and efficacious therapeutic, and could be used to treat patients suffering from B lymphocyte and antigen presenting cell driven autoimmune disease or undergoing allograft transplant where CD4O-CD154 interactions are involved in contributing to pathology.

    摘要翻译： 抗CD40抗体尚未报道在患者中诱导止血事件，但是接受抗CD40 Ab Chir12.12的B细胞淋巴瘤患者胰腺酶升高以及胰腺炎的可能风险可能导致使用此抗Fc抗体抗体， CD40抗体在慢性自身免疫性疾病和移植中的安全性原因。 因此，我们在体外和体内产生了不能介导抗体依赖性细胞毒性（ADCC）或补体依赖性细胞毒性（CDC）的Fc-沉默IgG1抗CD40抗体（mAb1，mAb2和mAb3）。 mAb1与亚治疗剂量的环孢菌素组合能够延长非人灵长类动物肾同种异体移植物存活。 此外，mAb1能够完全抑制用T细胞依赖性抗原免疫的一抗和二抗。 至关重要的是，在移植或免疫研究中没有止血事件或异常胰组织学的迹象。 总体而言，这些结果表明，mAb1将是一种安全有效的治疗方法，可用于治疗患有B淋巴细胞和抗原呈递细胞驱动的自身免疫性疾病或进行同种异体移植的患者，其中CD4O-CD154相互作用涉及病理学。

    Compositions and methods for inducing gene expression
    7.
    发明授权
    Compositions and methods for inducing gene expression 有权
    诱导基因表达的组合物和方法

    公开(公告)号：US06432927B1

    公开(公告)日：2002-08-13

    申请号：US09579897

    申请日：2000-05-26

    申请人： Richard J. Gregory ,  Karen Vincent

    发明人： Richard J. Gregory ,  Karen Vincent

    IPC分类号： A61K3170

    CPC分类号： C12N15/85 ,  A61K38/00 ,  A61K48/00 ,  C07K14/4702 ,  C07K14/52 ,  C07K2319/00 ,  C07K2319/71 ,  C07K2319/80 ,  C12N2799/022

    摘要： The present invention provides recombinant nucleic acid molecules encoding a chimeric transactivator protein including a DNA binding domain of a DNA binding protein and a protein domain capable of transcriptional activation. The present invention also provides recombinant viral and non-viral vectors that are able to infect and/or transfect and sustain expression of a biologically active chimeric transactivator proteins in mammalian cells. Also provided are host cell lines and non-human transgenic animals capable of expressing biologically active chimeric transactivator proteins. In another aspect, compositions and methods for treating or preventing ischemic damage associated with hypoxia-related disorders are provided.

    摘要翻译： 本发明提供了编码嵌合反式激活因子蛋白的重组核酸分子，其包括DNA结合蛋白的DNA结合结构域和能够转录激活的蛋白质结构域。 本发明还提供能够感染和/或转染和维持哺乳动物细胞中生物活性嵌合反式激活因子蛋白表达的重组病毒和非病毒载体。 还提供了能够表达生物活性嵌合反式激活蛋白的宿主细胞系和非人转基因动物。 在另一方面，提供了用于治疗或预防与缺氧相关疾病相关的缺血性损伤的组合物和方法。