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81.发明授权公开(公告)号：US09950011B1
公开(公告)日：2018-04-24
申请号：US15865075
申请日：2018-01-08
发明人： Derek Jantz , James Jefferson Smith , Michael G. Nicholson , Daniel T. MacLeod , Jeyaraj Antony , Victor Bartsevich
IPC分类号： A61K35/17 , C07K16/30 , C12N15/09 , A61K39/00 , A61K48/00 , C07K14/725 , C07K14/705 , C12N5/0783 , C12N15/113 , C12N15/86
CPC分类号： A61K35/17 , A61K39/0011 , A61K48/0008 , A61K2039/505 , A61K2039/5156 , A61K2039/5158 , C07K14/70503 , C07K14/7051 , C07K16/2803 , C07K16/30 , C07K16/3061 , C07K2317/53 , C07K2319/01 , C07K2319/02 , C07K2319/03 , C07K2319/33 , C07K2319/40 , C07K2319/74 , C12N5/0636 , C12N15/09 , C12N15/113 , C12N15/86 , C12N2510/00
摘要： Disclosed herein is a genetically-modified cell comprising in its genome a modified human T cell receptor alpha constant region gene, wherein the cell has reduced cell-surface expression of the endogenous T cell receptor. The present disclosure further relates to methods for producing such a genetically-modified cell, and to methods of using such a cell for treating a disease in a subject.
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82.发明授权公开(公告)号：US09434931B2
公开(公告)日：2016-09-06
申请号：US14858989
申请日：2015-09-18
发明人： James Jefferson Smith , Derek Jantz
CPC分类号： C12N9/22 , C12N9/16 , C12N15/8509 , C12N15/902 , C12N15/907 , C12N2800/80 , C12Y301/00 , C12Y301/04
摘要： Disclosed are rationally-designed, non-naturally-occurring meganucleases in which a pair of enzyme subunits having specificity for different recognition sequence half-sites are joined into a single polypeptide to form a functional heterodimer with a non-palindromic recognition sequence. The invention also relates to methods of producing such meganucleases, and methods of producing recombinant nucleic acids and organisms using such meganucleases.
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83.发明授权公开(公告)号：US09340777B2
公开(公告)日：2016-05-17
申请号：US14858986
申请日：2015-09-18
发明人： James Jefferson Smith , Derek Jantz
CPC分类号： C12N9/22 , C12N9/16 , C12N15/8509 , C12N15/902 , C12N15/907 , C12N2800/80 , C12Y301/00 , C12Y301/04
摘要： Disclosed are rationally-designed, non-naturally-occurring meganucleases in which a pair of enzyme subunits having specificity for different recognition sequence half-sites are joined into a single polypeptide to form a functional heterodimer with a non-palindromic recognition sequence. The invention also relates to methods of producing such meganucleases, and methods of producing recombinant nucleic acids and organisms using such meganucleases.
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84.发明公开公开(公告)号：US20240299585A1
公开(公告)日：2024-09-12
申请号：US18260578
申请日：2022-01-07
发明人： James Jefferson Smith , Janel Lape , John Morris
CPC分类号： A61K48/0058 , A61K48/0075 , A61P13/00 , C12N9/22 , C12N15/86 , C12N15/907 , C12Y101/03015 , C12N2750/14143
摘要： Disclosed are engineered meganucleases that bind and cleave a recognition sequence within a hydroxyacid oxidase 1 (HAO1) gene. The present invention also encompasses methods of using such engineered meganucleases to make genetically-modified cells. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or nucleic acids encoding engineered meganucleases of the invention, and the use of such compositions for treatment of primary hyperoxaluria type I (PH1).
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公开(公告)号：US12006522B2
公开(公告)日：2024-06-11
申请号：US18360312
申请日：2023-07-27
发明人： Gary Owens , Janel Lape , James Jefferson Smith , John Morris , Caitlin Turner , Whitney Lewis , Derek Jantz
CPC分类号： C12N9/22 , C12N15/86 , C12N2750/14143
摘要： The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.
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公开(公告)号：US20240084276A1
公开(公告)日：2024-03-14
申请号：US18516247
申请日：2023-11-21
CPC分类号： C12N9/22 , C12N15/86 , C12N2750/14143 , C12N2750/14145
摘要： Disclosed herein are recombinant meganucleases engineered to recognize and cleave a recognition sequence present in the human mitochondrial DNA (mtDNA). The disclosure further relates to the use of such recombinant meganucleases in methods for producing genetically-modified eukaryotic cells, and to a population of genetically-modified eukaryotic cells wherein the mtDNA has been having modified or edited.
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87.发明公开公开(公告)号：US20240002796A1
公开(公告)日：2024-01-04
申请号：US18155615
申请日：2023-01-17
发明人： Derek Jantz , James Jefferson Smith , Michael G. Nicholson , Daniel T. MacLeod , Jeyaraj Antony , Victor Bartsevich
IPC分类号： C12N5/0783 , A61K39/00 , A61K35/17 , C12N15/09 , C12N15/113 , C12N15/86 , A61K48/00 , C07K14/705 , C07K14/725 , C07K16/30 , C07K16/28
CPC分类号： C12N5/0636 , C07K2319/01 , A61K35/17 , C12N15/09 , C12N15/113 , C12N15/86 , A61K48/0008 , C07K14/70503 , C07K14/7051 , C07K16/30 , C07K16/3061 , C07K16/2803 , A61K2039/5156 , A61K2039/5158 , C12N2510/00 , A61K2039/505 , C07K2317/53 , C07K2319/02 , C07K2319/03 , C07K2319/33 , C07K2319/40 , C07K2319/74 , A61K39/0011
摘要： Disclosed herein is a genetically-modified cell comprising in its genome a modified human T cell receptor alpha constant region gene, wherein the cell has reduced cell-surface expression of the endogenous T cell receptor. The present disclosure further relates to methods for producing such a genetically-modified cell, and to methods of using such a cell for treating a disease in a subject.
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88.发明公开公开(公告)号：US20230416711A1
公开(公告)日：2023-12-28
申请号：US18185726
申请日：2023-03-17
发明人： James Jefferson Smith , Derek Jantz
CPC分类号： C12N9/22 , C12N15/902 , C12N9/16 , C12N15/8509 , C12N15/907 , C12N2800/80 , C12Y301/04 , C12Y301/00
摘要： Disclosed are rationally-designed, non-naturally-occurring meganucleases in which a pair of enzyme subunits having specificity for different recognition sequence half-sites are joined into a single polypeptide to form a functional heterodimer with a non-palindromic recognition sequence. The invention also relates to methods of producing such meganucleases, and methods of producing recombinant nucleic acids and organisms using such meganucleases.
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公开(公告)号：US20230183664A1
公开(公告)日：2023-06-15
申请号：US17998419
申请日：2021-05-10
发明人： Hui Li , James Jefferson Smith , Derek Jantz
CPC分类号： C12N9/22 , C12N15/86 , C12N2830/008 , C12N2830/42 , C12N2750/14143
摘要： Disclosed herein are viral vectors for use in recombinant molecular biology techniques. In particular, the present disclosure relates to self-limiting viral vectors containing nucleic acid sequences that encode engineered nucleases as well as nuclease recognition sequences such that expression of the engineered nuclease in a cell cleaves the viral vector and limits its persistence time. In some embodiments, the viral vectors disclosed herein also carry directives to delete, insert, or change a target sequence.
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公开(公告)号：US20220195407A1
公开(公告)日：2022-06-23
申请号：US17609244
申请日：2020-05-07
发明人： James Jefferson Smith , Hui Li
摘要： The invention provides engineered meganucleases, derived from I-Crel, which have substitutions at particular positions that increase the activity of the nucleases for recognition sequences containing certain center sequences. The invention also provides methods of cleaving double-stranded DNA using such engineered meganucleases. The invention further provides methods for improving the activity of engineered meganucleases for recognition sequences containing certain center sequences.
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