PLASMID FREE AAV VECTOR PRODUCING CELL LINES

    公开(公告)号:WO2019217483A1

    公开(公告)日:2019-11-14

    申请号:PCT/US2019/031209

    申请日:2019-05-07

    IPC分类号: C12N7/00 C12N7/04 C12N15/86

    摘要: Disclosed herein are packaging cell lines, in which adenovirus (Ad) E1A is constitutively expressed, that also contain integrated AAV rep and cap genes. The packaging cell lines exhibit little to no expressed Rep protein until helper virus function, such as adenovirus (Ad) E4, E2A and/or VA RNA are provided by, for example, transduction of the cells with a virus, vector or plasmid, such as an Ad-AAV hybrid virus. The promoter driving expression of AAV rep gene can be positioned far enough upstream (5') of the rep coding sequence that E1A is unable to activate the promoter, activate substantial transcription of the rep gene and in turn produce Rep protein. Introduction of helper virus function, such as E2A, E4 and/or VA RNA into these packaging cells is able to drive AAV rep gene transcription, subsequent Rep protein expression and production of rAAV vector particles.

    LIGHT INHIBITORS FOR SCLERODERMA AND SKIN FIBROTIC DISEASE TREATMENT
    5.
    发明申请
    LIGHT INHIBITORS FOR SCLERODERMA AND SKIN FIBROTIC DISEASE TREATMENT 审中-公开
    轻度抑制剂治疗沙眼肌病和皮肤纤维性疾病治疗

    公开(公告)号:WO2016127059A2

    公开(公告)日:2016-08-11

    申请号:PCT/US2016/016784

    申请日:2016-02-05

    IPC分类号: A61K39/395

    摘要: Methods of treating inflammatory conditions, disease and disorders of skin are provided. Methods include, for example, contacting or administering a sufficient amount of a LIGHT inhibitor to a subject to treat skin inflammation, skin fibrosis, or a skin fibrotic disease or disorder such as scleroderma, atopic dermatitis, nephrogenic fibrosing dermopathy, mixed connective tissue disease, scleromyxedema, scleredema, keloid, sclerodactyly, or eosinophilic fasciitis.

    摘要翻译: 提供了治疗炎性疾病,疾病和皮肤疾病的方法。 方法包括例如向受试者接触或施用足够量的LIGHT抑制剂以治疗皮肤炎症,皮肤纤维化或皮肤纤维化疾病或病症如硬皮病,特应性皮炎,肾性纤维化皮肤病,混合性结缔组织病, 硬皮病,水肿,瘢痕疙瘩,硬皮病或嗜酸粒细胞性筋膜炎。

    SPECIFIC AND UNIQUE T CELL RESPONSES AND MOLECULAR SIGNATURES FOR TREATMENT AND DIAGNOSIS OF MYCOBACTERIUM TUBERCULOSIS
    7.
    发明申请
    SPECIFIC AND UNIQUE T CELL RESPONSES AND MOLECULAR SIGNATURES FOR TREATMENT AND DIAGNOSIS OF MYCOBACTERIUM TUBERCULOSIS 审中-公开
    特异性和独特的T细胞应答和分子标记治疗和诊断MYCOBACTERIUM TUBCCOSOSIS

    公开(公告)号:WO2015157561A1

    公开(公告)日:2015-10-15

    申请号:PCT/US2015/025184

    申请日:2015-04-09

    IPC分类号: C12Q1/04

    摘要: The invention relates to novel targets for immune response modulation, treatment of tuberculosis infection and epitopes of Mycobacterium tuberculosis, or subsequences, portions or modifications thereof, and methods and compounds for treatment and prevention of tuberculosis infection. The invention is based, in part, on the discovery of novel Mycobacterium tuberculosis and non-tuberculosis mycobacterium T cell epitopes and use of such epitopes in treatment and vaccination methods. In particular embodiments, the invention provides proteins and peptides comprising amino acid sequences of Mycobacterium tuberculosis and non-tuberculosis mycobacterium proteins, and subsequences, portions or modifications, and methods and compounds comprising such protein and peptides for the treatment, diagnosis and prevention of Mycobacterium tuberculosis and non-tuberculosis mycobacterium infection.

    摘要翻译: 本发明涉及免疫应答调节,结核病感染治疗和结核分枝杆菌表位的新靶点,或其子序列,部分或修饰以及用于治疗和预防结核病感染的方法和化合物。 本发明部分地基于新型结核分枝杆菌和非结核分枝杆菌T细胞表位的发现以及在治疗和接种方法中使用这些表位。 在具体实施方案中,本发明提供包含结核分枝杆菌和非结核分枝杆菌蛋白质的氨基酸序列的蛋白质和肽,以及包含用于治疗,诊断和预防结核分枝杆菌的这些蛋白质和肽的亚序列,部分或修饰以及方法和化合物 和非结核分枝杆菌感染。

    SYNTHESIS OF BETA-TURN PEPTIDOMIMETIC CYCLIC COMPOUNDS
    10.
    发明申请
    SYNTHESIS OF BETA-TURN PEPTIDOMIMETIC CYCLIC COMPOUNDS 审中-公开
    β-环糊精环化合物的合成

    公开(公告)号:WO2013191926A1

    公开(公告)日:2013-12-27

    申请号:PCT/US2013/044829

    申请日:2013-06-07

    IPC分类号: C07K5/12 C07K1/107

    摘要: The present invention relates to methods of preparing β-turn cyclic peptidomimetic compounds and intermediates thereof. Particularly, the present invention relates to a process for the synthesis of β-turn cyclic peptidomimetic compounds of formula (I) where R 1 , R 2 , R 3 , R 4 , R 5 , R 6 , R 7 , L 2 , X, Y and n are as defined in the specification. The present invention provides a more efficient route for preparing β-turn cyclic peptidomimetic compounds and intermediates thereof.

    摘要翻译: 本发明涉及制备β-袢环肽模拟化合物的方法及其中间体。 特别地,本发明涉及合成式(I)的β-袢环肽模拟化合物的方法,其中R 1,R 2,R 3,R 4,R 5,R 6,R 7,L 2,X,Y和n如 规格。 本发明提供了一种更有效的制备β-袢环肽模拟化合物及其中间体的途径。