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1. 发明申请

US20110236353A1 ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR 审中-公开
标题翻译： ADENO相关病毒（AAV）分类，序列，包含它们的载体及其用途
公开(公告)号：US20110236353A1
公开(公告)日：2011-09-29
申请号：US13023918
申请日：2011-02-09
申请人： James M. Wilson , Guangping Gao , Mauricio R. Alvira , Luc H. Vandenberghe
发明人： James M. Wilson , Guangping Gao , Mauricio R. Alvira , Luc H. Vandenberghe
IPC分类号： A61K48/00 , C12N7/00 , C12N15/63 , C12N1/00 , C12N5/10 , C12N7/01 , A61K38/16 , A61P7/00 , A61P11/00
CPC分类号： A61K48/00 , C07K14/005 , C07K14/705 , C12N7/00 , C12N9/644 , C12N15/86 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14151 , C12N2830/008 , C12N2830/48 , C12N2830/85 , C12Y304/21022
摘要： Sequences of novel adeno-associated virus capsids and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.
摘要翻译：提供新的腺相关病毒衣壳和含有这些序列的载体和宿主细胞的序列。还描述了在生产rAAV颗粒中使用这种宿主细胞和载体的方法。还提供使用本发明的载体的AAV介导的治疗和免疫原性基因的递送。

2. 发明授权

US07906111B2 Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor 有权
标题翻译：腺相关病毒（AAV）进化枝，序列，含有其的载体及其用途
公开(公告)号：US07906111B2
公开(公告)日：2011-03-15
申请号：US10573600
申请日：2004-09-30
申请人： James M. Wilson , Guangping Gao , Mauricio R. Alvira , Luc H. Vandenberghe
发明人： James M. Wilson , Guangping Gao , Mauricio R. Alvira , Luc H. Vandenberghe
IPC分类号： C12N15/86 , C12N5/10 , C12N15/37 , C12N15/11 , C12N7/01
CPC分类号： A61K48/00 , C07K14/005 , C07K14/705 , C12N7/00 , C12N9/644 , C12N15/86 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14151 , C12N2830/008 , C12N2830/48 , C12N2830/85 , C12Y304/21022
摘要： Sequences of novel adeno-associated virus capsids and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.
摘要翻译：提供新的腺相关病毒衣壳和含有这些序列的载体和宿主细胞的序列。还描述了在生产rAAV颗粒中使用这种宿主细胞和载体的方法。还提供使用本发明的载体的AAV介导的治疗和免疫原性基因的递送。

3. 发明申请

US20130323226A1 Compositions and Methods for Altering Tissue Specificity and Improving AAV9-Mediated Gene Transfer 有权
标题翻译：改变组织特异性和改善AAV9介导的基因转移的组合物和方法
公开(公告)号：US20130323226A1
公开(公告)日：2013-12-05
申请号：US13985630
申请日：2012-02-17
申请人： James M. Wilson , Christie L. Bell , Luc H. Vandenberghe
发明人： James M. Wilson , Christie L. Bell , Luc H. Vandenberghe
IPC分类号： A61K31/7088 , A61K31/685 , A61K38/47
CPC分类号： A61K31/7088 , A61K31/685 , A61K38/47 , C12N15/86 , C12N2750/14043 , C12N2750/14045 , C12N2810/6027
摘要： A method of altering the targeting and/or cellular uptake efficiency of an adeno-associated virus (AAV) viral vector having a capsid containing an AAV9 cell surface binding domain is described. The method involves modifying a clade F cell surface receptor which comprises a glycan having a terminal sialic acid residue and a penultimate β-galactose residue. The modification may involve retargeting the vector by temporarily functionally ablate AAV9 binding in a subset of cells, thereby redirecting the vector to another subset of cells. Alternatively, the modification may involve increasing cellular update efficiency by treating the cells with a neuraminidase to expose cell surface β-galactose. Also provided are compositions containing the AAV9 vector and a neuraminidase. Also provided is a method for purifying AAV9 using β-galactose linked to solid support. Also provided are mutant vectors which have been modified to alter their targeting specificity, including mutant AAV9 in which the galactose binding domain is mutated and AAV in which an AAV9 galactose binding domain is engineered.
摘要翻译：描述了改变具有包含AAV9细胞表面结合结构域的衣壳的腺相关病毒（AAV）病毒载体的靶向和/或细胞摄取效率的方法。该方法包括修饰进化枝F细胞表面受体，其包含具有末端唾液酸残基的聚糖和倒数第二半乳糖残基。该修饰可能涉及通过临时功能地消融细胞子集中的AAV9结合来重新定向载体，从而将载体重定向到另一个细胞亚群。或者，修饰可以涉及通过用神经氨酸酶处理细胞以暴露细胞表面β-半乳糖来增加细胞更新效率。还提供了含有AAV9载体和神经氨酸酶的组合物。还提供了使用与固体支持物连接的β-半乳糖来纯化AAV9的方法。还提供已被修饰以改变其靶向特异性的突变载体，包括其中半乳糖结合结构域突变的突变AAV9和其中AAV9半乳糖结合结构域被工程化的AAV。

4. 发明申请

US20100260799A1 SIMIAN SUBFAMILY C ADENOVIRUSES SAdV-40, -31, AND -34 AND USES THEREOF 有权
标题翻译： SIMIAN SUBFAMILY C ADENOVIRUSES SAdV-40，-31和-34及其用途
公开(公告)号：US20100260799A1
公开(公告)日：2010-10-14
申请号：US12744405
申请日：2008-11-24
申请人： Soumitra Roy , James M. Wilson , Luc H. Vandenberghe
发明人： Soumitra Roy , James M. Wilson , Luc H. Vandenberghe
IPC分类号： A61K39/235 , C12N7/01 , C07H21/04 , C12N15/63 , C07K14/01 , C12N9/50 , A61P31/12
CPC分类号： C12N7/00 , C12N2710/10321 , C12N2710/10322 , C12N2710/10343
摘要： A recombinant vector comprises simian adenovirus SAdV-31 sequences and a heterologous gene under the control of regulatory sequences. A cell line which expresses simian adenovirus SAdV-31 gene(s) is also disclosed. Methods of using the vectors and cell lines are provided.
摘要翻译：重组载体包含猿猴腺病毒SAdV-31序列和在调控序列控制下的异源基因。也公开了表达猿猴腺病毒SAdV-31基因的细胞系。提供了使用载体和细胞系的方法。

5. 发明申请

US20090317417A1 Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof 审中-公开
标题翻译：具有降低衣壳免疫原性的改良的AAV载体及其用途
公开(公告)号：US20090317417A1
公开(公告)日：2009-12-24
申请号：US12226536
申请日：2007-04-27
申请人： Luc H. Vandenberghe , James M. Wilson
发明人： Luc H. Vandenberghe , James M. Wilson
IPC分类号： A61K39/235 , C12N7/00 , A61P31/12
CPC分类号： C12N15/86 , A61K2039/57 , C12N2750/14142 , C12N2750/14143
摘要： A method of reducing the cellular immune response and/or toxicity of AAV-mediated delivery is described. The method provides for masking or ablating a RxxR motif which induces T-cells, and which is located on select AAV capsids. The method further provides for reducing or eliminating heparin binding to an AAV. Also provided are compositions containing modified AAV capsids and methods of using same.
摘要翻译：描述了减少AAV介导的递送的细胞免疫应答和/或毒性的方法。该方法提供掩蔽或消融诱导T细胞的RxxR基序，其位于选定的AAV衣壳上。该方法进一步提供了减少或消除肝素与AAV的结合。还提供了含有修饰的AAV衣壳的组合物及其使用方法。

6. 发明授权

US09884071B2 Compositions and methods for altering tissue specificity and improving AAV9-mediated gene transfer 有权
公开(公告)号：US09884071B2
公开(公告)日：2018-02-06
申请号：US13985630
申请日：2012-02-17
申请人： James M. Wilson , Christie L. Bell , Luc H. Vandenberghe
发明人： James M. Wilson , Christie L. Bell , Luc H. Vandenberghe
IPC分类号： C12N15/86 , A61K48/00 , C07K14/005 , C12N7/00 , C12N15/85 , A61K31/7088 , A61K31/685 , A61K38/47
CPC分类号： A61K31/7088 , A61K31/685 , A61K38/47 , C12N15/86 , C12N2750/14043 , C12N2750/14045 , C12N2810/6027
摘要： A method of altering the targeting and/or cellular uptake efficiency of an adeno-associated virus (AAV) viral vector having a capsid containing an AAV9 cell surface binding domain is described. The method involves modifying a clade F cell surface receptor which comprises a glycan having a terminal sialic acid residue and a penultimate β-galactose residue. The modification may involve retargeting the vector by temporarily functionally ablate AAV9 binding in a subset of cells, thereby redirecting the vector to another subset of cells. Alternatively, the modification may involve increasing cellular update efficiency by treating the cells with a neuraminidase to expose cell surface β-galactose. Also provided are compositions containing the AAV9 vector and a neuraminidase. Also provided is a method for purifying AAV9 using β-galactose linked to solid support. Also provided are mutant vectors which have been modified to alter their targeting specificity, including mutant AAV9 in which the galactose binding domain is mutated and AAV in which an AAV9 galactose binding domain is engineered.

7. 发明申请

US20120189582A1 SIMIAN ADENOVIRUS 41 AND USES THEREOF 有权
标题翻译： SIMIAN ADENOVIRUS 41及其使用
公开(公告)号：US20120189582A1
公开(公告)日：2012-07-26
申请号：US13321985
申请日：2010-05-27
申请人： Soumitra Roy , James M. Wilson , Luc H. Vandenberghe
发明人： Soumitra Roy , James M. Wilson , Luc H. Vandenberghe
IPC分类号： A61K35/76 , A61K38/02 , C12N15/34 , C07K14/075 , C12N7/01 , C12N15/63
CPC分类号： C12N15/86 , A61K39/145 , A61K48/00 , A61K2039/5256 , C07K14/005 , C12N7/00 , C12N2710/10043 , C12N2710/10321 , C12N2710/10322 , C12N2710/10343 , C12N2810/6018
摘要： Novel simian adenovirus 41 and two isolates thereof are described. Various uses of these isolates, including construction of a recombinant vector which comprises simian adenovirus 41 sequences and a heterologous gene under the control of regulatory sequences are provided. A cell line which expresses simian adenovirus 41 gene(s) is also disclosed. Methods of using the vectors and cell lines are provided.
摘要翻译：描述了新型猿猴腺病毒41及其两个分离物。提供这些分离物的各种用途，包括构建包含猿猴腺病毒41序列和在调控序列控制下的异源基因的重组载体。还公开了表达猿腺病毒41基因的细胞系。提供了使用载体和细胞系的方法。

8. 发明授权

US08470310B2 Simian adenoviruses SAdV-36, -42.1, -42.2, and -44 and uses thereof 有权
标题翻译：猿猴腺病毒SAdV-36，-42.1，-42.2和-44及其用途
公开(公告)号：US08470310B2
公开(公告)日：2013-06-25
申请号：US12920648
申请日：2009-03-03
申请人： Soumitra Roy , James M. Wilson , Luc H. Vandenberghe
发明人： Soumitra Roy , James M. Wilson , Luc H. Vandenberghe
IPC分类号： A61K35/76 , C12N7/00
CPC分类号： C12N7/00 , A61K35/761 , A61K39/145 , A61K2039/5256 , A61K2039/57 , C07K14/005 , C12N15/86 , C12N2710/10321 , C12N2710/10322 , C12N2710/10343 , C12N2710/10371 , C12N2760/16122 , C12N2760/16134 , C12N2760/16171
摘要： A recombinant vector comprises simian adenovirus 36, simian adenovirus 42.1, simian adenovirus 42.2 and/or simian adenovirus 44 sequences and a heterologous gene under the control of regulatory sequences. A cell line which expresses one or more simian adenovirus-36, -42.1, -42.2 or -44 gene(s) is also described. Methods of using the vectors and cell lines are provided.
摘要翻译：重组载体包含猿猴腺病毒36，猿猴腺病毒42.1，猿腺病毒42.2和/或猿腺病毒44序列和在调节序列控制下的异源基因。还描述了表达一种或多种猿猴腺病毒-36，-42.1，-42.2或-44基因的细胞系。提供了使用载体和细胞系的方法。

9. 发明授权

US08231880B2 Simian subfamily C adenoviruses SAdV-40, -31, and -34 and uses thereof 有权
标题翻译：猿猴亚科C腺病毒SAdV-40,31和-34及其用途
公开(公告)号：US08231880B2
公开(公告)日：2012-07-31
申请号：US12744405
申请日：2008-11-24
申请人： Soumitra Roy , James M. Wilson , Luc H. Vandenberghe
发明人： Soumitra Roy , James M. Wilson , Luc H. Vandenberghe
IPC分类号： A61K39/235 , C12N15/00
CPC分类号： C12N7/00 , C12N2710/10321 , C12N2710/10322 , C12N2710/10343
摘要： A recombinant vector comprises simian adenovirus SAdV-31 sequences and a heterologous gene under the control of regulatory sequences. A cell line which expresses simian adenovirus SAdV-31 gene(s) is also disclosed. Methods of using the vectors and cell lines are provided.
摘要翻译：重组载体包含猿猴腺病毒SAdV-31序列和在调控序列控制下的异源基因。还公开了表达猿猴腺病毒SAdV-31基因的细胞系。提供了使用载体和细胞系的方法。

10. 发明申请

US20100247490A1 SIMIAN E ADENOVIRUSES SAdV-39, -25.2, -26, -30, -37, AND -38 有权
标题翻译： SIMIAN E ADENOVIRUSES SAdV-39，-25.2，-26，-30，-37和-38
公开(公告)号：US20100247490A1
公开(公告)日：2010-09-30
申请号：US12744441
申请日：2008-11-24
申请人： Soumitra Roy , James M. Wilson , Luc H. Vandenberghe
发明人： Soumitra Roy , James M. Wilson , Luc H. Vandenberghe
IPC分类号： A61K35/76 , C12N7/00 , C12N7/01 , C07H21/04 , C12N15/861 , C07K14/075 , A61K38/16 , A61P31/12 , C12N5/02
CPC分类号： C12N15/86 , A61K48/00 , A61K2039/525 , A61K2039/5256 , C07K14/005 , C12N7/00 , C12N2710/10321 , C12N2710/10322 , C12N2710/10343 , C12N2710/10361 , C12N2760/16122
摘要： A recombinant vector comprises simian adenovirus SAdV-39, -25.2, -26, -30, -37, and -38 sequences and a heterologous gene under the control of regulatory sequences. A cell line which expresses simian adenovirus SAdV-39, -25.2, -26, -30, -37, and -383 gene(s) is also disclosed. Methods of using the vectors and cell lines are provided.
摘要翻译：重组载体包含猿猴腺病毒SAdV-39，-25.2，-26，-30，-37和-38序列，以及在调控序列控制下的异源基因。还公开了表达猿猴腺病毒SAdV-39，-25.2，-26，-30，-37和-383基因的细胞系。提供了使用载体和细胞系的方法。

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