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1. 发明授权

US06281010B1 Adenovirus gene therapy vehicle and cell line 失效
标题翻译：腺病毒基因治疗载体和细胞系
公开(公告)号：US06281010B1
公开(公告)日：2001-08-28
申请号：US08549489
申请日：1995-10-27
申请人： Guang-Ping Gao , James M. Wilson
发明人： Guang-Ping Gao , James M. Wilson
IPC分类号： C12N1500
CPC分类号： C12N15/86 , A61K48/00 , C12N2710/10343 , C12N2750/14143 , Y10S977/802 , Y10S977/907
摘要： A novel adenovirus E1/E4 expressing packaging cell line is provided, which permits the generation of recombinant adenoviruses deleted in both gene regions. The E1/E4 deleted recombinant adenovirus is capable of expressing a selected transgene product in cells in vivo or in vitro. This recombinant virus is useful in the treatment of genetic disorders.
摘要翻译：提供了表达新型腺病毒E1 / E4的包装细胞系，其能够在两个基因区域中产生缺失的重组腺病毒。 E1 / E4缺失的重组腺病毒能够在体内或体外在细胞中表达所选择的转基因产物。这种重组病毒可用于治疗遗传疾病。

2. 发明授权

US5756283A Method for improved production of recombinant adeno-associated viruses for gene therapy 失效
标题翻译：通过重组腺相关病毒改善转导的方法
公开(公告)号：US5756283A
公开(公告)日：1998-05-26
申请号：US462014
申请日：1995-06-05
申请人： James M. Wilson , Krishna J. Fisher , Guang-Ping Gao
发明人： James M. Wilson , Krishna J. Fisher , Guang-Ping Gao
IPC分类号： A61K48/00 , C12N15/864 , C12Q1/70 , C12N7/04 , C12N13/00 , C12N15/00
CPC分类号： C12N15/86 , A61K48/00 , C12N2750/14143
摘要： A method for enhancing the efficiency of transduction of a recombinant AAV into a target cell is provided. The target cell is infected with a recombinant adeno-associated virus comprising a selected transgene under the control of regulatory sequences. The infected cell is contacted with an agent which facilitates the conversion of single stranded recombinant virus to its double stranded form. When this conversion occurs in the target cell, enhanced transduction of the recombinant virus into said target cell results. The agent can be a helper virus providing a gene which facilitates the conversion, or an agent to which the infected cell is exposed, which facilitates the conversion. In a similar manner, a novel recombinant AAV is provided which contains the facilitating gene and the transgene. The methods may be performed ex vivo or in vivo.
摘要翻译：提供了一种用于提高重组AAV转导入靶细胞的效率的方法。靶细胞用包含选择的转基因的重组腺相关病毒感染，在调控序列的控制下。将感染的细胞与促进单链重组病毒转化为其双链形式的试剂接触。当转化发生在靶细胞中时，将重组病毒增强转导到所述靶细胞中。该试剂可以是提供有助于转化的基因的辅助病毒，或感染细胞暴露于其中的试剂，其有助于转化。以类似的方式，提供了含有促进基因和转基因的新型重组AAV。该方法可以离体或体内进行。

3. 发明授权

US06270996B1 Recombinant adenovirus and adeno-associated virus, cell lines and methods of production and use thereof 失效
标题翻译：重组腺病毒和腺相关病毒，细胞系及其生产和使用方法
公开(公告)号：US06270996B1
公开(公告)日：2001-08-07
申请号：US09563869
申请日：2000-05-03
申请人： James M. Wilson , Krishna J. Fisher , Guang-Ping Gao
发明人： James M. Wilson , Krishna J. Fisher , Guang-Ping Gao
IPC分类号： C12N510
CPC分类号： C12N15/86 , A61K48/00 , C12N2710/10343 , C12N2750/14143 , Y10S977/802 , Y10S977/907
摘要： An adenovirus E1/E4 expressing packaging cell line is provided, which permits the generation of recombinant adenoviruses deleted in both gene regions. A method for enhancing the efficiency of transduction of a recombinant AAV into a target cell is provided by infecting a target cell with a recombinant AAV comprising a selected transgene under the control of regulatory sequences. The infected cell is contacted with an agent which facilitates the conversion of single stranded recombinant virus to its double stranded form.
摘要翻译：提供了表达腺病毒E1 / E4的包装细胞系，其允许在两个基因区域中产生缺失的重组腺病毒。通过在包含调节序列控制下的选择的转基因的重组AAV感染靶细胞来提供将重组AAV转导进靶细胞的效率的方法。将感染的细胞与促进单链重组病毒转化为其双链形式的试剂接触。

4. 发明授权

US06261551B1 Recombinant adenovirus and adeno-associated virus, cell lines, and methods of production and use thereof 失效
标题翻译：重组腺病毒和腺相关病毒，细胞系及其生产和使用方法
公开(公告)号：US06261551B1
公开(公告)日：2001-07-17
申请号：US08973334
申请日：1997-12-05
申请人： James M. Wilson , Krishna J. Fisher , Guang-Ping Gao
发明人： James M. Wilson , Krishna J. Fisher , Guang-Ping Gao
IPC分类号： A61K4800
CPC分类号： C12N15/86 , A61K48/00 , C12N2710/10343 , C12N2750/14143 , Y10S977/802 , Y10S977/907
摘要： An adenovirus E1/E4 expressing packaging cell line is provided, which permits the generation of recombinant adenoviruses deleted in both gene regions. A method for enhancing the efficiency of transduction of a recombinant AAV into a target cell is provided by infecting a target cell with a recombinant AAV comprising a selected transgene under the control of regulatory sequences. The infected cell is contacted with an agent which facilitates the conversion of single stranded recombinant virus to its double stranded form.
摘要翻译：提供了表达腺病毒E1 / E4的包装细胞系，其允许在两个基因区域中产生缺失的重组腺病毒。通过在包含调节序列控制下的选择的转基因的重组AAV感染靶细胞来提供将重组AAV转导进靶细胞的效率的方法。将感染的细胞与促进单链重组病毒转化为其双链形式的试剂接触。

5. 发明授权

US06258595B1 Compositions and methods for helper-free production of recombinant adeno-associated viruses 有权
标题翻译：无辅助生产重组腺相关病毒的组合物和方法
公开(公告)号：US06258595B1
公开(公告)日：2001-07-10
申请号：US09404555
申请日：1999-09-23
申请人： Guang-Ping Gao , James M. Wilson
发明人： Guang-Ping Gao , James M. Wilson
IPC分类号： C12N1586
CPC分类号： G01R31/275 , C12N15/86 , C12N2750/14143 , C12N2750/14151 , C12N2840/20 , G01R31/013 , G01R31/316
摘要： A method for producing recombinant adeno-associated virus in the absence of contaminating helper virus or wild-type virus involves culturing a mammalian host cell containing an rAd/AAV hybrid virus, an AAV rep sequence and an AAV cap sequence under the control of regulatory sequences directing expression thereof. The rAd/AAV hybrid virus contains a rAAV construct to be packaged into an AAV virion in a backbone containing the adenoviral sequences necessary to express E1a and E1b gene products and to permit replication of the hybrid virus. The method of the invention permits replication of the hybrid virus and production of rAAV virion in this host cell in the absence of a helper virus and obviates a subsequent purification step to purify rAAV from contaminating virus.
摘要翻译：在不存在污染性辅助病毒或野生型病毒的情况下用于产生重组腺相关病毒的方法包括在调节序列的控制下培养含有rAd / AAV杂合病毒，AAV rep序列和AAV帽序列的哺乳动物宿主细胞指导其表达。 rAd / AAV杂合病毒含有一个rAAV构建体，其被包装在含有表达E1a和E1b基因产物所必需的腺病毒序列并且允许杂交病毒复制的骨架中的AAV病毒粒子中。本发明的方法允许在不存在辅助病毒的情况下复制该杂种病毒并在该宿主细胞中产生rAAV病毒粒子，并且消除随后的纯化步骤以从污染病毒纯化rAAV。

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