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公开(公告)号：US20220062437A1
公开(公告)日：2022-03-03
申请号：US17230354
申请日：2021-04-14
摘要： A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. A method of treating an ocular disease, e.g., Stargardt's Disease, caused by a defect or mutation in a target gene, e.g., ABCA4 comprising: administering to the ocular cells of a subject having an ocular disease a composition comprising a recombinant AAV comprising a nucleic acid trans-splicing molecule as described above.
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公开(公告)号：US20180369412A1
公开(公告)日：2018-12-27
申请号：US15776663
申请日：2016-11-18
摘要： A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. A method of treating an ocular disease, e.g., Stargardt's Disease, caused by a defect or mutation in a target gene, e.g., ABCA4 comprising: administering to the ocular cells of a subject having an ocular disease a composition comprising a recombinant AAV comprising a nucleic acid trans-splicing molecule as described above.
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公开(公告)号：US10987433B2
公开(公告)日：2021-04-27
申请号：US15776663
申请日：2016-11-18
摘要： A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. A method of treating an ocular disease, e.g., Stargardt's Disease, caused by a defect or mutation in a target gene, e.g., ABCA4 comprising: administering to the ocular cells of a subject having an ocular disease a composition comprising a recombinant AAV comprising a nucleic acid trans-splicing molecule as described above.
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公开(公告)号：US20240218394A1
公开(公告)日：2024-07-04
申请号：US18504261
申请日：2023-11-08
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：US11993776B2
公开(公告)日：2024-05-28
申请号：US17047496
申请日：2019-04-17
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 , C12Q1/68
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：US20230233709A1
公开(公告)日：2023-07-27
申请号：US18061633
申请日：2022-12-05
发明人： Jean Bennett , Jeannette Bennicelli , Junwei Sun , Ji Yun Song , Sergei Nikonov
CPC分类号： A61K48/005 , A61P27/02 , C12N15/86 , A61K48/0075 , C12N2750/14122 , C12N2750/14143 , C12N2800/22
摘要： Compositions and methods are provided for treating Leber congenital amaurosis (LCA) in a subject. In one aspect, a recombinant adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding Lebercilin. In another aspect, Lebercilin has an amino acid sequence of SEQ ID NO: 1. In yet another aspect, the nucleic acid molecule has a sequence of SEQ ID NO: 3 or a variant thereof. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.
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公开(公告)号：US20230009257A1
公开(公告)日：2023-01-12
申请号：US17822533
申请日：2022-08-26
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：US20220204989A1
公开(公告)日：2022-06-30
申请号：US17604228
申请日：2020-04-17
发明人： Krishna J. Fisher , Jean Bennett
摘要： A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. The trans-splicing molecule includes a 3′ transcription terminator domain which enhances the efficiency of trans-splicing. The 3′ TTD comprises a triple helix domain and a tRNA-like domain.
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公开(公告)号：US11206977B2
公开(公告)日：2021-12-28
申请号：US15524126
申请日：2015-11-04
发明人： Jean Bennett
摘要： The present invention provides a reading test to measure vision loss. In one embodiment, the vision loss is due to disease progression. The tests are useful in evaluating the effects of intervention in vision deterioration. The tests are non-invasive, simple, quick, sensitive, reproducible, and easy to administer. The tests measure the subject's reading speed and accuracy under defined conditions of illumination and contrast. The results of these tests may be used to determine if treatment of a disease should be initiated, terminated, altered, or remain unchanged.
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公开(公告)号：US10668129B2
公开(公告)日：2020-06-02
申请号：US15576027
申请日：2016-05-20
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE-CNRS , UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6) , THE REGENTS OF THE UNIVERSITY OF CALIFORNIA , THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： Thierry Leveillard , John Flannery , Xin Mei , Leah Byrne , José-Alain Sahel , Emmanuelle Clerin-Lachapelle , Junwei Sun , Jean Bennett , Jeannette Bennicelli
摘要： The present invention relates to the synergistic combination of the short and long Rod-Derived Cone Viability Factors and to methods for treating retinal degenerative diseases.
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