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1. 发明申请

WO2005005610A3 MUTANT ADENO-ASSOCIATED VIRUS VIRIONS AND METHODS OF USE THEREOF 审中-公开
标题翻译：突变腺病毒相关病毒血症及其使用方法
公开(公告)号：WO2005005610A3
公开(公告)日：2005-12-15
申请号：PCT/US2004021121
申请日：2004-06-29
申请人： UNIV CALIFORNIA , SCHAFFER DAVID V , KASPAR BRIAN , MAHESHRI NARENDRA
发明人： SCHAFFER DAVID V , KASPAR BRIAN , MAHESHRI NARENDRA
IPC分类号： C07H21/04 , C07K1/00 , C07K14/015 , C12N20060101 , C12N7/00 , C12N7/01 , C12N7/02 , C12N15/00 , C12N15/09 , C12N15/63 , C12N15/70 , C12N15/74 , C12N15/864 , C12Q1/68 , C12Q1/70
CPC分类号： C07K14/005 , C12N15/86 , C12N2750/14122 , C12N2750/14143
摘要： The present invention provides mutant adeno-associated virus (AAV) that exhibit altered capsid properties, e.g., reduced binding to neutralizing antibodies in serum and/or altered heparin binding and/or altered infectivity of particular cell types. The present invention further provides libraries of mutant AAV comprising one or more mutations in a capsid gene. The present invention further provides methods of generating the mutant AAV and mutant AAV libraries, and compositions comprising the mutant AAV. The present invention further provides recombinant AAV (rAAV) virions that comprise a mutant capsid protein. The present invention further provides nucleic acids comprising nucleotide sequences that encode mutant capsid proteins, and host cells comprising the nucleic acids. The present invention further provides methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.
摘要翻译：本发明提供突变型腺相关病毒（AAV），其显示改变的衣壳性质，例如减少与血清中的中和抗体的结合和/或肝素结合的变化和/或特定细胞类型的改变的感染性。本发明还提供了在衣壳基因中包含一个或多个突变的突变AAV的文库。本发明还提供了产生突变AAV和突变AAV文库的方法，以及包含突变体AAV的组合物。本发明还提供了包含突变型衣壳蛋白的重组AAV（rAAV）病毒粒子。本发明还提供了包含编码突变衣壳蛋白的核苷酸序列的核酸和包含核酸的宿主细胞。本发明进一步提供将基因产物递送给个体的方法，所述方法通常涉及向有需要的个体施用有效量的受试者rAAV病毒粒子。

2. 发明申请

WO2005005610A2 MUTANT ADENO-ASSOCIATED VIRUS VIRIONS AND METHODS OF USE THEREOF 审中-公开
标题翻译：突变腺病毒相关病毒及其使用方法
公开(公告)号：WO2005005610A2
公开(公告)日：2005-01-20
申请号：PCT/US2004/021121
申请日：2004-06-29
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA , SCHAFFER, David, V. , KASPAR, Brian , MAHESHRI, Narendra
发明人： SCHAFFER, David, V. , KASPAR, Brian , MAHESHRI, Narendra
IPC分类号： C12N
CPC分类号： C07K14/005 , C12N15/86 , C12N2750/14122 , C12N2750/14143
摘要： The present invention provides mutant adeno-associated virus (AAV) that exhibit altered capsid properties, e.g., reduced binding to neutralizing antibodies in serum and/or altered heparin binding and/or altered infectivity of particular cell types. The present invention further provides libraries of mutant AAV comprising one or more mutations in a capsid gene. The present invention further provides methods of generating the mutant AAV and mutant AAV libraries, and compositions comprising the mutant AAV. The present invention further provides recombinant AAV (rAAV) virions that comprise a mutant capsid protein. The present invention further provides nucleic acids comprising nucleotide sequences that encode mutant capsid proteins, and host cells comprising the nucleic acids. The present invention further provides methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.
摘要翻译：本发明提供了表现出改变的衣壳特性（例如与血清中的中和抗体的结合减少和/或改变的肝素结合和/或特定细胞类型的感染性改变）的突变腺伴随病毒（AAV）。本发明进一步提供了在衣壳基因中包含一个或多个突变的突变AAV的文库。本发明进一步提供了产生突变AAV和突变AAV文库的方法，以及包含突变AAV的组合物。本发明进一步提供了包含突变体衣壳蛋白的重组AAV（rAAV）病毒粒子。本发明进一步提供了包含编码突变体衣壳蛋白的核苷酸序列的核酸，以及包含该核酸的宿主细胞。本发明进一步提供了将基因产物递送至个体的方法，所述方法通常包括将有效量的受试者rAAV病毒体给予有需要的个体。

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