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公开(公告)号：US09233131B2
公开(公告)日：2016-01-12
申请号：US12277164
申请日：2008-11-24
IPC分类号： C12N15/861 , A61K39/12 , A61K35/76 , A61K31/7088 , A61K38/17 , C07K14/005 , C12N7/00 , C12N15/86 , A61K48/00
CPC分类号： A61K35/76 , A61K31/7088 , A61K38/1709 , A61K48/00 , C07K14/005 , C12N7/00 , C12N15/86 , C12N2750/14121 , C12N2750/14122 , C12N2750/14123 , C12N2750/14132 , C12N2750/14143 , C12N2750/14145 , C12N2750/14151 , C12N2750/14161 , C12N2810/6027 , A61K2300/00
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。
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公开(公告)号：US20050053922A1
公开(公告)日：2005-03-10
申请号：US10880297
申请日：2004-06-28
申请人： David Schaffer , Brian Kaspar , Narendra Maheshri
发明人： David Schaffer , Brian Kaspar , Narendra Maheshri
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。
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公开(公告)号：US09441244B2
公开(公告)日：2016-09-13
申请号：US10880297
申请日：2004-06-28
IPC分类号： C12N15/86 , C07K14/005
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.
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公开(公告)号：US20070003524A1
公开(公告)日：2007-01-04
申请号：US11442504
申请日：2006-05-25
申请人： Brian Kaspar , Fred Gage , Jeffrey Rothstein
发明人： Brian Kaspar , Fred Gage , Jeffrey Rothstein
IPC分类号： A61K48/00 , C12N15/861
CPC分类号： C12N15/86 , A61K48/00 , A61K48/0075 , C12N2750/14143
摘要： Methods are disclosed for delivering a heterologous gene to a cell body of a neuron by contacting a muscle tissue innervated by the neuron with a viral vector comprising a heterologous gene, wherein the viral vector enters said neuron and is retrogradely moved to the cell body. Additionally, methods for expressing secreted proteins from a nerve cell body as well as methods for treating neurodegenerative disorders such as amyotrophic lateral sclerosis are described.
摘要翻译： 公开了通过使由神经元支配的肌肉组织与包含异源基因的病毒载体接触将异源基因递送至神经元的细胞体的方法，其中病毒载体进入所述神经元并逆行移动至细胞体。 此外，描述了用于从神经细胞体表达分泌蛋白的方法以及用于治疗神经变性疾病如肌萎缩性侧索硬化的方法。
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公开(公告)号：US20090202490A1
公开(公告)日：2009-08-13
申请号：US12277164
申请日：2008-11-24
CPC分类号： A61K35/76 , A61K31/7088 , A61K38/1709 , A61K48/00 , C07K14/005 , C12N7/00 , C12N15/86 , C12N2750/14121 , C12N2750/14122 , C12N2750/14123 , C12N2750/14132 , C12N2750/14143 , C12N2750/14145 , C12N2750/14151 , C12N2750/14161 , C12N2810/6027 , A61K2300/00
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。
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