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公开(公告)号：US20240318186A1
公开(公告)日：2024-09-26
申请号：US18673713
申请日：2024-05-24
发明人： Philip R. JOHNSON , Bruce C. Schnepp , Jean Bennett , Scott J. Dooley , Krishna Jawaharlal Fisher , Junwei Sun
IPC分类号： C12N15/113 , C07K14/705
CPC分类号： C12N15/1138 , C07K14/705 , C07K2319/71 , C07K2319/85 , C12N2320/33 , C12N2320/34
摘要： The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.
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公开(公告)号：US11879133B2
公开(公告)日：2024-01-23
申请号：US16607834
申请日：2018-04-24
CPC分类号： C12N15/86 , A61K9/0048 , A61P27/02 , A61K48/00 , C12N2710/10043
摘要： Compositions and methods are provided for treating ocular neuropathy in a subject. In one aspect, a recombinant adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding NRF2. In another aspect, a recombinant adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding SIRT1. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.
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公开(公告)号：US11793887B2
公开(公告)日：2023-10-24
申请号：US16615941
申请日：2018-05-31
申请人： The Trustees of the University of Pennsylvania , University of Southern California , The Research Institute of the McGill University Health Centre
发明人： Nancy Braverman , Catherine Argyriou , Joseph Hacia , Jean Bennett , Junwei Sun , Ji Yun Song , Devin McDougald
IPC分类号： A61K48/00 , C12N15/79 , C07H21/04 , A61K35/761 , C12N9/14 , C12N15/86 , C12N15/864
CPC分类号： A61K48/005 , A61K35/761 , C12N9/14 , C12N15/86 , C07H21/04 , C12N15/8645 , C12N2750/14143 , C12N2800/22
摘要： Methods and compositions are provided for treatment of peroxisomal biogenesis disorders (PBDs). More particularly, recombinant adeno-associated viruses (rAAV) provided in the form of compositions are used to deliver a nucleic acid encoding human PEX1 to host cells. The rAAVs comprise a AAV capsid, and packaged therein a vector genome comprising an AAV 5′ inverted terminal repeat (ITR) sequence; a promoter; a coding sequence encoding a human PEX1; and an AAV 3′ ITR.
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公开(公告)号：US20220118111A1
公开(公告)日：2022-04-21
申请号：US17569896
申请日：2022-01-06
发明人： Jean Bennett , Junwei Sun , Vidyullatha Vasireddy
摘要： Codon optimized nucleic acid sequences for RDH12 are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequence for functional RDH12. Recombinant vectors are provided that express the codon optimized, functional RDH12. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence, deficiency or inappropriate expression of RDH12. Other compositions and methods are providing for correcting a non-functional, defective or inadequately expressed native RDH12.
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公开(公告)号：US11197936B2
公开(公告)日：2021-12-14
申请号：US16314179
申请日：2017-07-07
发明人： Jean Bennett , Junwei Sun , Vidyullatha Vasireddy
摘要： Codon optimized nucleic acid sequences for RDH12 are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequence for functional RDH12. Recombinant vectors are provided that express the codon optimized, functional RDH12. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence, deficiency or inappropriate expression of RDH12. Other compositions and methods are providing for correcting a non-functional, defective or inadequately expressed native RDH12.
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公开(公告)号：US20190062385A1
公开(公告)日：2019-02-28
申请号：US16180892
申请日：2018-11-05
发明人： Theodore G. Drivas , Jean Bennett
IPC分类号： C07K14/47
摘要： Compositions are provided that comprise a recombinant vector carrying a nucleic acid sequence encoding a fragment of CEP290 lacking all or part of its N-terminal and C-terminal inhibitory regions, under the control of regulatory sequences which express the product of said gene in a selected cell of a mammalian subject, and a pharmaceutically acceptable carrier. These and other compositions are disclosed with are useful in methods for treating a mammalian subject having a disease associated with a CEP290 mutation, such as Lebers Congenital Amaurosis.
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公开(公告)号：US20180369415A1
公开(公告)日：2018-12-27
申请号：US16066970
申请日：2017-01-05
发明人： Jeannette Bennicelli , Jean Bennett , Junwei Sun
摘要： Codon optimized nucleic acid sequences for the long form and short form of RdCVF are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequences. Recombinant vectors are provided that express the codon optimized RdCVFL and RdCVF individually, or express two copies of a codon optimized RdCVF or RdCVFL nucleic acid sequence, or both RdCVFL and RdCVF in a single vector or virus. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence or inappropriate expression of RdCVF and RdCVFL.
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公开(公告)号：US10155794B2
公开(公告)日：2018-12-18
申请号：US14904447
申请日：2014-07-11
发明人： Theodore G. Drivas , Jean Bennett
IPC分类号： C07K14/47 , C12Q1/6883 , A61K38/00 , A61K48/00
摘要： Compositions are provided that comprise a recombinant vector carrying a nucleic acid sequence encoding a fragment of CEP290 lacking all or part of its N-terminal and C-terminal inhibitory regions, under the control of regulatory sequences which express the product of said gene in a selected cell of a mammalian subject, and a pharmaceutically acceptable carrier. These and other compositions are disclosed with are useful in methods for treating a mammalian subject having a disease associated with a CEP290 mutation, such as Lebers Congenital Amaurosis.
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公开(公告)号：US09896665B2
公开(公告)日：2018-02-20
申请号：US14979786
申请日：2015-12-28
IPC分类号： C12N7/00 , C12N15/86 , C07K14/435
CPC分类号： C12N7/00 , C07K14/435 , C12N15/86 , C12N2750/14111 , C12N2750/14121 , C12N2750/14141 , C12N2750/14143 , C12N2750/14152 , C12N2800/107 , C12N2830/60 , C12N2830/90
摘要： Proviral plasmids contain a modular gene expression cassette with one or a combination of (i) a wildtype 5′ AAV2 ITR sequence flanked by unique restriction sites that permit ready removal or replacement of the ITR; (ii) a promoter flanked by unique restriction sites that permit ready removal or replacement of the entire promoter sequence; (iii) a polylinker sequence that permits insertion of a gene coding sequence without modification thereof, wherein the gene is operatively linked to, and under the regulatory control of, the aforementioned promoter; (iv) a bovine growth hormone polyadenylation sequence flanked by unique restriction sites that permit ready removal or replacement of the polyA sequence; and (v) a wildtype 3′ AAV2 ITR sequence flanked by unique restriction sites that permit ready removal or replacement of the 3′ ITR. These plasmids enable rapid manipulation of the components of the cassette, such as rapid mutation and/or replacement of any component, and thereby increase the efficiency of recombinant viral vector, such as rAAV, production.
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公开(公告)号：US20170049910A1
公开(公告)日：2017-02-23
申请号：US15246001
申请日：2016-08-24
IPC分类号： A61K48/00 , C12N15/86 , C07K14/705 , A61K38/17 , C07K14/005 , C12N7/00
CPC分类号： C12N15/86 , A61K38/177 , A61K48/0058 , A61K48/0075 , C07K14/005 , C07K14/015 , C07K14/723 , C12N7/00 , C12N15/8645 , C12N2750/14121 , C12N2750/14122 , C12N2750/14143 , C12N2830/008
摘要： Described herein are capsid proteins and adeno-associated viruses capable of targeting various types of ocular cells including bipolar and horizontal cells. Also described herein are methods of treating various ocular disorders in a subject in need thereof by administering to the subject an effective concentration of a composition comprising the recombinant adeno-associated virus (AAV) of the invention.
摘要翻译： 本文描述了能够靶向各种类型的眼细胞（包括双极细胞和水平细胞）的衣壳蛋白和腺相关病毒。 本文还描述了通过向受试者施用包含本发明的重组腺相关病毒（AAV）的组合物的有效浓度来治疗有需要的受试者中的各种眼部疾病的方法。
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