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公开(公告)号：US11767346B2
公开(公告)日：2023-09-26
申请号：US17689324
申请日：2022-03-08
IPC分类号： C07K14/005 , C12N15/10 , C12N15/86 , C12N7/00
CPC分类号： C07K14/005 , C12N15/1037 , C12N15/86 , C12N7/00 , C12N2320/32 , C12N2750/14011 , C12N2750/14121 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145
摘要： Adeno associated viral (AAV) particles are emerging as a useful vehicle for gene delivery to various organs and tissues, one of them being the retina. Provided here are variant AAV (for example, variant serotype 2 (AAV2)) capsid proteins and variant capsid protein containing particles with enhanced ability to transduce retinal cells.
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公开(公告)号：US20220267383A1
公开(公告)日：2022-08-25
申请号：US17689324
申请日：2022-03-08
IPC分类号： C07K14/005 , C12N15/10 , C12N15/86
摘要： Adeno associated viral (AAV) particles are emerging as a useful vehicle for gene delivery to various organs and tissues, one of them being the retina. Provided here are variant AAV (e.g., variant serotype 2 (AAV2)) capsid proteins and variant capsid protein containing particles with enhanced ability to transduce retinal cells.
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公开(公告)号：US20210061863A1
公开(公告)日：2021-03-04
申请号：US17009536
申请日：2020-09-01
发明人： Sergei Zolotukhin , Sanford L. Boye , Shannon E. Boye , Damien Marsic , Nicholas Muzyczka , Hector Ruben Mendez-Gomez , Paul E. Gamlin
IPC分类号： C07K14/005 , C12N15/10 , C12N15/86
摘要： Adeno associated viral (AAV) particles are emerging as a useful vehicle for gene delivery to various organs and tissues, one of them being the retina. Provided here are variant AAV (e.g., variant serotype 2 (AAV2)) capsid proteins and variant capsid protein containing particles with enhanced ability to transduce retinal cells.
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公开(公告)号：US11332502B2
公开(公告)日：2022-05-17
申请号：US17009536
申请日：2020-09-01
发明人： Sergei Zolotukhin , Sanford L. Boye , Shannon E. Boye , Damien Marsic , Nicholas Muzyczka , Hector Ruben Mendez-Gomez , Paul D. Gamlin
IPC分类号： C07K14/005 , C12N15/10 , C12N15/86 , C12N7/00
摘要： Adeno associated viral (AAV) particles are emerging as a useful vehicle for gene delivery to various organs and tissues, one of them being the retina. Provided here are variant AAV (e.g., variant serotype 2 (AAV2)) capsid proteins and variant capsid protein containing particles with enhanced ability to transduce retinal cells.
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公开(公告)号：US10793606B2
公开(公告)日：2020-10-06
申请号：US16547532
申请日：2019-08-21
IPC分类号： C07K14/005 , C12N15/10 , C12N15/86 , C12N7/00
摘要： Adeno associated viral (AAV) particles are emerging as useful vehicle for gene delivery to various organs and tissues, one of them being the retina. Provided here are variant AAV (e.g., variant serotype 2 (AAV2)) capsid proteins and variant capsid protein containing particles with enhanced ability to transduce retinal cells.
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公开(公告)号：US20200002386A1
公开(公告)日：2020-01-02
申请号：US16547532
申请日：2019-08-21
发明人： Sergei Zolotukhin , Sanford L. Boye , Shannon E. Boye , Damien Marsic , Nicholas Muzyczka , Hector Ruben Mendez-Gomez , Paul D. Gamlin
IPC分类号： C07K14/005 , C12N15/86 , C12N15/10
摘要： Adeno associated viral (AAV) particles are emerging as useful vehicle for gene delivery to various organs and tissues, one of them being the retina. Provided here are variant AAV (e.g., variant serotype 2 (AAV2)) capsid proteins and variant capsid protein containing particles with enhanced ability to transduce retinal cells.
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7.发明申请公开(公告)号：US20180223312A1
公开(公告)日：2018-08-09
申请号：US15824023
申请日：2017-11-28
发明人： Arun Srivastava , Li Zhong , Sergei Zolotukhin , George Vladimirovich Aslanidi , Mavis Agbandje-McKenna , Kim M. Van Vliet , Chen Ling
IPC分类号： C12N15/86
摘要： Disclosed are next-generation multi-mutated capsid protein-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using these high transduction efficiency vector constructs in a variety of therapeutic applications including, inter alia, as delivery agents for the treatment or amelioration of one or more diseases or abnormal conditions in an affected mammal using in vivo and/or ex situ viral vector-based gene therapy protocols. Also disclosed are large-scale production methods for the multi-mutated, capsid-modified rAAV expression vectors, viral particles, and infectious virions, as well as use of the disclosed compositions in the manufacture of medicaments for use in a variety of in vitro and/or in vivo therapeutic methodologies.
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公开(公告)号：US20230203533A1
公开(公告)日：2023-06-29
申请号：US17922174
申请日：2021-04-28
CPC分类号： C12N15/86 , C40B10/00 , C12N2710/14143 , C12N2750/14151 , C12N2750/14143
摘要： Provided herein are methods and compositions useful in the screening and production of recombinant AAV (rAAV) that have structural fitness and potency in insect cells and mammalian cells. The disclosure provides a directed evolution system for generating and selecting rAAV variants based on their Kozak sequences. In some embodiments, methods disclosed herein comprise the use of modified Kozak sequences to express AAV2 VP1 proteins in amounts that are useful for producing rAAV particles having high infectivities and/or transduction efficiencies. In some embodiments, methods of producing, and methods of screening, rAAV are provided that comprise the steps of infecting insect cells by administering rAAV to these cells, recovering rAAV-integrated DNA from these insect cells, and transfecting recovered rAAV into mammalian cells. Also provided herein are nucleic acids comprising nucleotide sequences encoding novel Kozak sequences. Further provided herein are mammalian cells, baculovirus particles and insect cells comprising these nucleic acids.
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公开(公告)号：US10723768B2
公开(公告)日：2020-07-28
申请号：US15680668
申请日：2017-08-18
发明人： Li Zhong , Sergei Zolotukhin , Lakshmanan Govindasamy , Mavis Agbandje-McKenna , Arun Srivastava
IPC分类号： C07K14/005 , C12N15/86 , A61K48/00 , A61K39/00 , C12N15/864 , A61K35/76 , C12N7/00
摘要： Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.
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10.发明授权公开(公告)号：US11702673B2
公开(公告)日：2023-07-18
申请号：US16657959
申请日：2019-10-18
IPC分类号： C12N15/86 , C12N7/00 , C07K14/075 , C12N5/07
CPC分类号： C12N15/86 , C07K14/075 , C12N5/0601 , C12N7/00 , C12N2710/14041 , C12N2710/14143 , C12N2710/14144 , C12N2750/14123 , C12N2750/14143 , C12N2750/14151
摘要： Provided herein are methods and compositions useful in the production of recombinant AAV (rAAV) in host producer cells, such as insect cells. In some embodiments, methods, uses and compositions are provided that comprise recombinant VP1 genes comprising modified Kozak sequences to express AAV VP1 proteins in amounts that are useful for producing infective rAAV particles. These infective rAAV particles may comprise a gene of interest.
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