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公开(公告)号：US20200263201A1
公开(公告)日：2020-08-20
申请号：US16667608
申请日：2019-10-29
IPC分类号： C12N15/86
摘要： A recombinant vector comprises simian adenovirus 43, 45, 46, 47, 48, 49 or 50 sequences and a heterologous gene under the control of regulatory sequences. A cell line which expresses simian adenovirus 43, 45, 46, 47, 48, 49 or 50 gene(s) is also disclosed. Methods of using the vectors and cell lines are provided.
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32.发明授权公开(公告)号：US10406173B2
公开(公告)日：2019-09-10
申请号：US15862999
申请日：2018-01-05
IPC分类号： A61K48/00 , C12N15/85 , C07K14/005 , A61K9/127 , C07K14/47 , A61K31/7088 , A61K31/685 , A61K38/47 , C12N15/86
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.
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33.发明授权公开(公告)号：US10265417B2
公开(公告)日：2019-04-23
申请号：US15227418
申请日：2016-08-03
IPC分类号： C12N15/864 , C12N7/00 , C12N15/37 , C12N15/11 , A61K48/00 , A61K39/42 , C07K14/005 , C12N15/86 , C07K14/705 , C12N9/64
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.
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公开(公告)号：US20190055523A1
公开(公告)日：2019-02-21
申请号：US16179322
申请日：2018-11-02
IPC分类号： C12N7/00 , C07K14/005 , A61K48/00
摘要： A method for producing AAV, without requiring cell lysis, is described. The method involves harvesting AAV from the supernatant. For AAV having capsids with a heparin binding site, the method involves modifying the AAV capsids and/or the culture conditions to ablate the binding between the AAV heparin binding site and the cells, thereby allowing the AAV to pass into the supernatant, i.e., media. Thus, the method of the invention provides supernatant containing high yields of AAV which have a higher degree of purity from cell membranes and intracellular materials, as compared to AAV produced using methods using a cell lysis step.
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35.发明申请公开(公告)号：US20190054188A1
公开(公告)日：2019-02-21
申请号：US16149218
申请日：2018-10-02
IPC分类号： A61K48/00 , C07K14/005 , C12N7/00 , C07K14/705 , C12N9/64 , C12N15/86
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.
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