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公开(公告)号:US20230272424A1
公开(公告)日:2023-08-31
申请号:US18165138
申请日:2023-02-06
发明人: Luk Vandenberghe , Guangping Gao , James M. Wilson
IPC分类号: C12N15/86 , A61K48/00 , C07K14/005 , C12N7/00
CPC分类号: C12N15/86 , A61K48/005 , A61K48/0091 , C07K14/005 , C12N7/00 , A61K48/00 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14152
摘要: A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.
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公开(公告)号:US11680274B2
公开(公告)日:2023-06-20
申请号:US16368758
申请日:2019-03-28
发明人: Luk Vandenberghe , Guangping Gao , James M. Wilson
IPC分类号: C12N15/00 , C12N15/86 , A61K48/00 , C07K14/005 , C12N7/00
CPC分类号: C12N15/86 , A61K48/005 , A61K48/0091 , C07K14/005 , C12N7/00 , A61K48/00 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14152
摘要: A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.
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公开(公告)号:US20230340532A1
公开(公告)日:2023-10-26
申请号:US18061000
申请日:2022-12-02
发明人: Luk Vandenberghe , Guangping Gao , James M. Wilson
IPC分类号: C12N15/86 , A61K48/00 , C07K14/005 , C12N7/00
CPC分类号: C12N15/86 , A61K48/0091 , C07K14/005 , C12N7/00 , A61K48/005 , C12N2750/14122 , C12N2750/14143 , A61K48/00 , C12N2750/14142 , C12N2750/14152
摘要: A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.
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公开(公告)号:US10947561B2
公开(公告)日:2021-03-16
申请号:US16887833
申请日:2020-05-29
发明人: Luk Vandenberghe , Guangping Gao , James M. Wilson
IPC分类号: C12N15/00 , C12N15/86 , A61K48/00 , C07K14/005 , C12N7/00
摘要: A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.
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公开(公告)号:US20230416780A1
公开(公告)日:2023-12-28
申请号:US18345183
申请日:2023-06-30
发明人: Luk Vandenberghe , Guangping Gao , James M. Wilson
IPC分类号: C12N15/86 , A61K48/00 , C07K14/005 , C12N7/00
CPC分类号: C12N15/86 , A61K48/0091 , C07K14/005 , C12N7/00 , A61K48/005 , C12N2750/14122 , C12N2750/14143 , A61K48/00 , C12N2750/14142 , C12N2750/14152
摘要: A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.
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公开(公告)号:US10392622B2
公开(公告)日:2019-08-27
申请号:US15500591
申请日:2015-07-31
IPC分类号: C12N15/63
摘要: A self-regulating gene expression construct comprises a single promoter in operative association with a repressor sequence (e.g., bacterial repressor lacI or gaiR), operator sequence(s) responsive to the expressed repressor protein, and a transgene. A dual-regulating construct comprises a single promoter controlling expression of a bacterial repressor sequence and a transgene, and which, in the presence of a first inducer molecule, transcribes the transgene and repressor; and a ribozyme in association with an aptamer sequence, the aptamer sequence capable of interacting with a second inducer molecule to terminate mRNA degradation by the ribozyme. Also provided are recombinant vectors or viruses containing the self-regulating or dual self-regulating constructs and cells containing the vectors. Such compositions are useful in methods of treating a diseases using gene therapy.
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公开(公告)号:US10301648B2
公开(公告)日:2019-05-28
申请号:US14624671
申请日:2015-02-18
发明人: Luk Vandenberghe , Guangping Gao , James M. Wilson
IPC分类号: C12N15/86 , A61K48/00 , C07K14/005 , C12N7/00
摘要: A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.
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公开(公告)号:US20230287452A1
公开(公告)日:2023-09-14
申请号:US18054786
申请日:2022-11-11
发明人: Luk Vandenberghe , Guangping Gao , James M. Wilson
IPC分类号: C12N15/86 , A61K48/00 , C07K14/005 , C12N7/00
CPC分类号: C12N15/86 , A61K48/0091 , C07K14/005 , C12N7/00 , A61K48/005 , C12N2750/14122 , C12N2750/14143 , A61K48/00 , C12N2750/14142 , C12N2750/14152
摘要: A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.
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公开(公告)号:US20170191079A1
公开(公告)日:2017-07-06
申请号:US15408567
申请日:2017-01-18
发明人: Luk Vandenberghe , Guangping Gao , James M. Wilson
CPC分类号: C12N15/86 , A61K48/00 , A61K48/005 , A61K48/0091 , C07K14/005 , C12N7/00 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14152
摘要: A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.
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