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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：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：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：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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