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公开(公告)号：EP4403642A2
公开(公告)日：2024-07-24
申请号：EP24175675.8
申请日：2018-04-03
发明人： TAGLIATELA, Stephanie , YOUNG, Andrew , CHEN, Szu-Ying , RAMAMOORTHI, Kartik , OBERKOFLER, David
IPC分类号： C12Q1/68
CPC分类号： A01K2217/05220130101 , A01K2217/1520130101 , A01K2227/10520130101 , A01K2267/031220130101 , C07K14/705 , C12N15/86 , A61K48/0058 , C12N2840/00720130101 , A61P25/28 , A61P25/08 , A61K48/00 , C12N2750/1414320130101
摘要： Provided herein are compositions and methods for selective expression of a transgene. Compositions and methods for selective expression of a transgene comprise one or more human regulatory elements, which, when operably linked to a transgene, can facilitate selective expression of a transgene (e.g., cell-type selective expression) in a target cell as compared to at least one or more non-target cells.
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公开(公告)号：EP3976798A1
公开(公告)日：2022-04-06
申请号：EP20815541.6
申请日：2020-05-29
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公开(公告)号：EP3717505A1
公开(公告)日：2020-10-07
申请号：EP18884285.0
申请日：2018-11-30
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公开(公告)号：EP4257155A3
公开(公告)日：2024-02-28
申请号：EP23185569.3
申请日：2019-11-15
发明人： RAMAMOORTHI, Kartik , TAGLIATELA, Stephanie , TANENHAUS, Anne , YOUNG, Andrew , CHEN, Szu-Ying , ZHANG, Chi , MARTIN, Stephanie , OBERKOFLER, David , WONG, Victoria , LI, Jianmin
摘要： Provided herein are nucleic acid molecules and vectors comprising variant copper-transporting ATPase 2 (ATP7B) nucleic acid sequences. Such sequences have been optimized for expression in mammalian cells, liver cells, and/or from an adeno associated viral vector (AAV), including truncated and/or codon optimized variants. Also provided are viral vectors comprising such ATP7B variant nucleic acid sequences, and methods of use thereof for treating disorders associated with an ATP7B deficiency, such as Wilson's disease.
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公开(公告)号：EP3607073A1
公开(公告)日：2020-02-12
申请号：EP18781685.5
申请日：2018-04-03
发明人： TAGLIATELA, Stephanie , YOUNG, Andrew , CHEN, Szu-Ying , RAMAMOORTHI, Kartik , OBERKOFLER, David
IPC分类号： C12N15/63 , A01K67/027 , C12Q1/68
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公开(公告)号：EP4403642A3
公开(公告)日：2024-10-09
申请号：EP24175675.8
申请日：2018-04-03
发明人： TAGLIATELA, Stephanie , YOUNG, Andrew , CHEN, Szu-Ying , RAMAMOORTHI, Kartik , OBERKOFLER, David
IPC分类号： C12N15/63 , A01K67/027 , C12Q1/68
CPC分类号： A01K2217/05220130101 , A01K2217/1520130101 , A01K2227/10520130101 , A01K2267/031220130101 , C07K14/705 , C12N15/86 , A61K48/0058 , C12N2840/00720130101 , A61P25/28 , A61P25/08 , A61K48/00 , C12N2750/1414320130101
摘要： Provided herein are compositions and methods for selective expression of a transgene. Compositions and methods for selective expression of a transgene comprise one or more human regulatory elements, which, when operably linked to a transgene, can facilitate selective expression of a transgene (e.g., cell-type selective expression) in a target cell as compared to at least one or more non-target cells.
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公开(公告)号：EP4257155A2
公开(公告)日：2023-10-11
申请号：EP23185569.3
申请日：2019-11-15
发明人： RAMAMOORTHI, Kartik , TAGLIATELA, Stephanie , TANENHAUS, Anne , YOUNG, Andrew , CHEN, Szu-Ying , ZHANG, Chi , MARTIN, Stephanie , OBERKOFLER, David , WONG, Victoria , LI, Jianmin
IPC分类号： A61K48/00
摘要： Provided herein are nucleic acid molecules and vectors comprising variant copper-transporting ATPase 2 (ATP7B) nucleic acid sequences. Such sequences have been optimized for expression in mammalian cells, liver cells, and/or from an adeno associated viral vector (AAV), including truncated and/or codon optimized variants. Also provided are viral vectors comprising such ATP7B variant nucleic acid sequences, and methods of use thereof for treating disorders associated with an ATP7B deficiency, such as Wilson's disease.
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公开(公告)号：EP3880809A1
公开(公告)日：2021-09-22
申请号：EP19884231.2
申请日：2019-11-15
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公开(公告)号：EP3624858A1
公开(公告)日：2020-03-25
申请号：EP18803184.3
申请日：2018-05-18
发明人： RAMAMOORTHI, Kartik , TAGLIATELA, Stephanie , TANENHAUS, Anne , YOUNG, Andrew , CHEN, Szu-Ying , ZHANG, Chi , MARTIN, Stephanie , OBERKOFLER, David
IPC分类号： A61K48/00 , C12N15/85 , C12N15/861
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