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1. 发明申请

WO2002085287A2 MINIMAL ADENOVIRAL VECTORS FOR IMMUNIZATION 审中-公开
标题翻译：最小的ADENOVIRAL矢量免疫
公开(公告)号：WO2002085287A2
公开(公告)日：2002-10-31
申请号：PCT/US2002/012237
申请日：2002-04-18
申请人： GENSTAR THERAPEUTICS CORP. , ZHANG, Wei-Wei , JOSEPHS, Steven , BALAGUE, Cristina , FANG, Xiangming
发明人： ZHANG, Wei-Wei , JOSEPHS, Steven , BALAGUE, Cristina , FANG, Xiangming
IPC分类号： A61K
CPC分类号： A01K67/0278 , A01K67/0275 , A01K67/0276 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2217/072 , A01K2217/075 , A01K2217/20 , A01K2227/105 , A01K2267/01 , A01K2267/0306 , A01K2267/0331 , A61K38/37 , A61K48/00 , C12N15/8509 , C12N15/86 , C12N2710/10343 , C12N2750/14143 , C12N2800/108 , C12N2800/30 , C12N2830/006 , C12N2830/008 , C12N2830/42 , C12N2830/85 , C12N2840/20 , C12N2840/203
摘要： The present invention provides a method for treating a disorder such as hemophilia. A method of treating hemophilia in a mammal by administering recombinant virus virions comprising a nucleotide sequence having an adenoviral inverted terminal repeat fusion sequence, a packaging signal, a transcriptional control region, and a nucleic acid encoding a therapeutic protein such as FVIII. In addition, the DNA molecule does not encode an adenoviral protein. It is preferred that the virions be administered to the mammal under conditions that result in the expression of the therapeutic protein at a level that provides a therapeutic effect in said mammal.
摘要翻译：本发明提供了治疗诸如血友病的病症的方法。通过施用包含具有腺病毒反向末端重复融合序列，包装信号，转录控制区和编码治疗性蛋白如FVIII的核酸的核苷酸序列的重组病毒颗粒来治疗哺乳动物血友病的方法。此外，DNA分子不编码腺病毒蛋白。优选地，在导致治疗性蛋白质表达在所述哺乳动物中提供治疗效果的水平的条件下，向哺乳动物施用病毒粒子。

2. 发明申请

WO2002085287A3 MINIMAL ADENOVIRAL VECTORS FOR IMMUNIZATION 审中-公开
公开(公告)号：WO2002085287A3
公开(公告)日：2002-10-31
申请号：PCT/US2002/012237
申请日：2002-04-18
申请人： GENSTAR THERAPEUTICS CORP. , ZHANG, Wei-Wei , JOSEPHS, Steven , BALAGUE, Cristina , FANG, Xiangming
发明人： ZHANG, Wei-Wei , JOSEPHS, Steven , BALAGUE, Cristina , FANG, Xiangming
IPC分类号： C12N15/00
摘要： The present invention provides a method for treating a disorder such as hemophilia. A method of treating hemophilia in a mammal by administering recombinant virus virions comprising a nucleotide sequence having an adenoviral inverted terminal repeat fusion sequence, a packaging signal, a transcriptional control region, and a nucleic acid encoding a therapeutic protein such as FVIII. In addition, the DNA molecule does not encode an adenoviral protein. It is preferred that the virions be administered to the mammal under conditions that result in the expression of the therapeutic protein at a level that provides a therapeutic effect in said mammal.

3. 发明申请

WO9745550A3 MINI-ADENOVIRAL VECTOR 审中-公开
标题翻译：微型腺病毒载体
公开(公告)号：WO9745550A3
公开(公告)日：1998-04-09
申请号：PCT/US9710218
申请日：1997-05-30
申请人： BAXTER INT , ZHANG WEI WEI , ALEMANY RAMON , DAI YIFAN , JOSEPHS STEVEN , BALAGUE CRISTINA , AYARES DAVID , SCHNEIDERMAN RICHARD
发明人： ZHANG WEI-WEI , ALEMANY RAMON , DAI YIFAN , JOSEPHS STEVEN , BALAGUE CRISTINA , AYARES DAVID , SCHNEIDERMAN RICHARD
IPC分类号： A01K67/027 , A61K31/711 , A61K35/76 , A61K38/00 , A61K38/43 , A61K48/00 , A61P7/04 , C07K14/755 , C12N5/10 , C12N7/00 , C12N15/09 , C12N15/34 , C12N15/85 , C12N15/861 , C12Q1/02 , C12Q1/68 , G01N21/78 , G01N33/53 , G01N33/68 , C12N15/86 , C12N7/01
CPC分类号： C12N15/8509 , A01K67/0278 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2217/075 , A01K2217/20 , A01K2267/0306 , A61K38/00 , A61K48/00 , C07K14/755 , C12N15/86 , C12N2710/10343 , C12N2800/108 , C12N2800/30 , C12N2810/859 , C12N2830/003 , C12N2830/008 , C12N2830/38 , C12N2830/85 , C12N2840/20 , C12N2840/203 , C12N2840/206
摘要： This invention is related to adenoviral (Ad) vectors and their applications in the field of genetic medicine, including gene transfer, gene therapy, and gene vaccination. More specifically, this invention is related to the Ad vectors that carry the minimal cis-element of the Ad genome (mini-Ad vector) and are capable of delivering transgenes and/or heterologous DNA up to 36 kb. The generation and propagation of the mini-Ad vectors require trans-complementation of a packaging-attenuated and replication-defective helper Ad (helper) in an Ad helper cell line. This invention further comprises a methodology for generating a mini-adenoviral (mini-Ad) vector for use in gene therapy of hemophilia and animal test systems for in vivo evaluation of the Ad vectors. More specifically, this invention describes factor VIII (FVIII) Ad vectors that only contain minimal cis-element of the Ad genome (so-called mini-Ad) and comprise a human FVIII cDNA with other supporting DNA elements up to 36 kb. The FVIII mini-Ad can be generated and preferentially amplified through the assistance of a packaging-attenuated helper Ad and a helper cell line. This invention also reports designs and methods for producing transgenic mouse models that can be used for in vivo testing the mini-Ad.

4. 发明申请

WO2003093486A1 IN SITU GENERATION OF VIRAL VECTORS 审中-公开
标题翻译：在病毒生成的病毒载体中
公开(公告)号：WO2003093486A1
公开(公告)日：2003-11-13
申请号：PCT/US2003/013503
申请日：2003-04-29
申请人： MAGNUM THERAPEUTICS , FITCHMUN, Mark , HUANG, Lei , RAHMAN, Amena , JOSEPHS, Steven , SOBOL, Robert, E.
发明人： FITCHMUN, Mark , HUANG, Lei , RAHMAN, Amena , JOSEPHS, Steven , SOBOL, Robert, E.
IPC分类号： C12N15/86
CPC分类号： C12N15/86 , A61K39/00 , A61K48/00 , C12N2710/10343 , C12N2830/008
摘要： Materials and methods useful in the in situ generation of viral vectors are described. Briefly, virus is liberated from cells by treatment with polysorbate and tri-butyl phosphate yielding non-viscous lysates with relatively little cell debris. Lysis is followed by step density ultracentrifugation to isolate crude virus. Finally, purification and helper removal is accomplished by dynamic banding ultracentrifugation.
摘要翻译：描述了用于原位产生病毒载体的材料和方法。简而言之，通过用聚山梨酸酯和磷酸三丁酯处理，从细胞中释放病毒，产生具有相对较少细胞碎片的非粘稠裂解物。裂解之后是步进密度超速离心以分离粗病毒。最后，通过动态条带超离心实现净化和辅助去除。

5. 发明申请

WO0208436A3 MINI-ADENOVIRAL VECTOR SYSTEM FOR VACCINATION 审中-公开
标题翻译：用于疫苗接种的微型腺病毒载体系统
公开(公告)号：WO0208436A3
公开(公告)日：2003-02-27
申请号：PCT/US0123005
申请日：2001-07-20
申请人： GENSTAR THERAPEUTICS CORP , ZHANG WEI-WEI , ALEMANY RAMON , DAI YIFAN , JOSEPHS STEVEN , BALAGUE CRISTINA , AYARES DAVID , SCHNEIDERMAN RICHARD
发明人： ZHANG WEI-WEI , ALEMANY RAMON , DAI YIFAN , JOSEPHS STEVEN , BALAGUE CRISTINA , AYARES DAVID , SCHNEIDERMAN RICHARD
IPC分类号： A61K39/00 , A61K48/00 , C07K14/755 , C12N15/12 , C12N15/16 , C12N15/19 , C12N15/861
CPC分类号： C12N15/86 , A61K39/00 , A61K48/00 , A61K2039/5256 , C07K14/755 , C12N2710/10343 , C12N2800/108 , C12N2800/30 , C12N2810/859 , C12N2830/008 , C12N2830/42 , C12N2830/85 , C12N2840/203 , C12N2840/206
摘要： The principle of this invention is to produce mini-Ad vectors using packaging-attenuated and replication-defective helper Ad and an E1-complementing Ad helper cell line. Since the essential cis-acting elements for Ad DNA replication and packaging are located at the ends of the viral genome (ITRs plus the packaging signal, less than 1 kb), the backbone of the mini-Ad vectors is trimmed down to contain only the essential cis -elements. The remainder of the Ad genome is to be replaced by non-viral DNA for gene transfer, retention, and expression. The capacity of the mini-Ad vectors may be up to 36 kb. The viral proteins required for DNA replication and encapsidation of the mini-Ad vectors are designed to be provided in trans from the helper Ad ( trans )-complementation) and the helper cell line. In order to generate relatively pure preparations of the mini-Ad virions, packaging of the helper Ad genome is controlled by selective attenuation of the packaging signal while that of the mini-Ad vector genome proceeds normally. In the absence of mini-Ad vector genome, the helper Ad does replicate and is propagated in an inefficient manner. In this system, the Ad helper cell line is necessary for the E1-complementation of the helper virus and may also provide other functions for packaging attenuation of the helper viral genome and enhancement of mini-Ad vector replication. If the preparation of mini-Ad vectors is not sufficiently pure, biochemical or other physical methods will be utilized to achieve further purification.
摘要翻译：本发明的原理是使用包装减毒和复制缺陷型辅助Ad和E1互补的Ad辅助细胞系来生产小型Ad载体。由于用于Ad DNA复制和包装的基本顺式作用元件位于病毒基因组的末端（ITRs加上包装信号小于1kb），微型Ad载体的主链被修剪成仅包含重要的顺式元件。 Ad基因组的其余部分将被用于基因转移，保留和表达的非病毒DNA替代。小型Ad载体的容量可达36kb。设计mini-Ad载体的DNA复制和壳化所需的病毒蛋白质是从辅助Ad（反式） - 互补）和辅助细胞系反向提供的。为了产生相对纯的微型Ad病毒粒子的制剂，通过选择性衰减包装信号来控制辅助Ad基因组的包装，而迷你Ad载体基因组的包装正常进行。在没有mini-Ad载体基因组的情况下，辅助Ad不会复制并以低效的方式传播。在该系统中，Ad辅助细胞系对辅助病毒的E1互补是必需的，并且还可以提供用于包装辅助病毒基因组衰减和增强小型Ad载体复制的其他功能。如果微型Ad载体的制备不够纯，将采用生物化学或其他物理方法来进一步纯化。

6. 发明申请

WO1997045550A2 MINI-ADENOVIRAL VECTOR 审中-公开
标题翻译：微型腺病毒载体
公开(公告)号：WO1997045550A2
公开(公告)日：1997-12-04
申请号：PCT/US1997010218
申请日：1997-05-30
申请人： BAXTER INTERNATIONAL INC. , ZHANG, Wei-Wei , ALEMANY, Ramon , DAI, Yifan , JOSEPHS, Steven , BALAGUE, Cristina , AYARES, David , SCHNEIDERMAN, Richard
发明人： BAXTER INTERNATIONAL INC.
IPC分类号： C12N15/86
CPC分类号： C12N15/8509 , A01K67/0278 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2217/075 , A01K2217/20 , A01K2267/0306 , A61K38/00 , A61K48/00 , C07K14/755 , C12N15/86 , C12N2710/10343 , C12N2800/108 , C12N2800/30 , C12N2810/859 , C12N2830/003 , C12N2830/008 , C12N2830/38 , C12N2830/85 , C12N2840/20 , C12N2840/203 , C12N2840/206
摘要： This invention is related to adenoviral (Ad) vectors and their applications in the field of genetic medicine, including gene transfer, gene therapy, and gene vaccination. More specifically, this invention is related to the Ad vectors that carry the minimal cis-element of the Ad genome (mini-Ad vector) and are capable of delivering transgenes and/or heterologous DNA up to 36 kb. The generation and propagation of the mini-Ad vectors require trans-complementation of a packaging-attenuated and replication-defective helper Ad (helper) in an Ad helper cell line. This invention further comprises a methodology for generating a mini-adenoviral (mini-Ad) vector for use in gene therapy of hemophilia and animal test systems for in vivo evaluation of the Ad vectors. More specifically, this invention describes factor VIII (FVIII) Ad vectors that only contain minimal cis-element of the Ad genome (so-called mini-Ad) and comprise a human FVIII cDNA with other supporting DNA elements up to 36 kb. The FVIII mini-Ad can be generated and preferentially amplified through the assistance of a packaging-attenuated helper Ad and a helper cell line. This invention also reports designs and methods for producing transgenic mouse models that can be used for in vivo testing the mini-Ad.
摘要翻译：本发明涉及腺病毒（Ad）载体及其在遗传医学领域的应用，包括基因转移，基因治疗和基因疫苗接种。更具体地，本发明涉及携带Ad基因组（mini-Ad载体）的最小顺式元件的Ad载体，并能够将转基因和/或异源DNA递送至36kb。迷你Ad载体的产生和繁殖需要在Ad辅助细胞系中进行包装减毒和复制缺陷型辅助Ad（辅助细胞）的互补。本发明还包括用于产生用于血友病的基因治疗的微型腺病毒（小型-Ad）载体和用于Ad载体的体内评估的动物测试系统的方法。更具体地，本发明描述了仅包含Ad基因组的最小顺式元件（所谓的mini-Ad）的因子VIII（FVIII）Ad载体，并且包含具有高达36kb的其他支持DNA元件的人FVIII cDNA。 FVIII mini-Ad可以通过包装衰减的辅助Ad和辅助细胞系的帮助产生并优先放大。本发明还报道了可用于体内测试小型Ad的转基因小鼠模型的设计和方法。

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