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公开(公告)号:US11857642B2
公开(公告)日:2024-01-02
申请号:US17155743
申请日:2021-01-22
CPC分类号: A61K48/0058 , A61K48/00 , C12N15/63 , C12N15/79 , C12N15/86 , C12N2750/14143 , C12N2830/008
摘要: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 150 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in rod photoreceptors of a gene when operatively linked to a nucleic acid sequence coding for said gene.
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公开(公告)号:US11739349B2
公开(公告)日:2023-08-29
申请号:US16764306
申请日:2018-11-15
发明人: Josephine Juettner , Jacek Krol , Botond Roska
IPC分类号: C12N15/86 , A61K38/17 , A61K48/00 , C07K14/705
CPC分类号: C12N15/86 , A61K38/177 , A61K48/0058 , C07K14/705 , C12N2750/14143 , C12N2830/008 , C12N2830/15
摘要: The present invention to a method for expressing an exogenous gene specifically in cells of the retinal pigment epithelium of a primate, this method comprising the step of delivering an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1, or consisting of a nucleic acid sequence of at least 400 bp having at least 80% overall identity to the sequence of SEQ ID NO:1, to cells of the retinal pigment epithelium of the primate, wherein this isolated nucleic acid molecule specifically leads to the expression of an exogenous gene in cells of the retinal pigment epithelium of primates when a nucleic acid sequence coding for the exogenous gene is operatively linked to this isolated nucleic acid molecule.
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公开(公告)号:US11591615B2
公开(公告)日:2023-02-28
申请号:US16768057
申请日:2018-11-28
发明人: Josephine Juettner , Jacek Krol , Botond Roska
IPC分类号: C12N15/86
摘要: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 1000 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in cells of the retinal pigment epithelium of a gene when operatively linked to a nucleic acid sequence coding for said gene.
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公开(公告)号:US11371060B2
公开(公告)日:2022-06-28
申请号:US16484252
申请日:2018-02-05
发明人: Botond Roska , Josephine Juettner
摘要: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 1800 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in retinal ganglion cells of a gene when operatively linked to a nucleic acid sequence coding for said gene.
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公开(公告)号:US11931427B2
公开(公告)日:2024-03-19
申请号:US17084963
申请日:2020-10-30
发明人: Josephine Juettner , Dasha Nelidova , Botond Roska
CPC分类号: A61K48/0075 , A61F9/0017 , A61K9/0048 , A61K48/005 , C07K14/705 , C12N15/85 , A61K38/00 , C12N2750/14143 , C12N2830/008
摘要: The present inventions relates to an isolated nucleic acid molecule comprising a nucleotide sequence coding for a depolarizing light-gated ion channel functionally linked to a promoter leading to the specific expression of said depolarizing light-gated ion channel in a retinal photoreceptor, or the nucleotide sequence complementary to said nucleotide sequence, for use in treating or ameliorating blindness. The present invention also relates to methods of using such nucleic acid molecules in the treatment of blindness.
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公开(公告)号:US11591618B2
公开(公告)日:2023-02-28
申请号:US17223875
申请日:2021-04-06
IPC分类号: C12N15/79 , A61K48/00 , C12N15/86 , C07K14/195 , C07K14/405
摘要: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 400 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in rod photoreceptors of a gene when operatively linked to a nucleic acid sequence coding for said gene.
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公开(公告)号:US10857241B2
公开(公告)日:2020-12-08
申请号:US15760207
申请日:2016-09-13
发明人: Josephine Juettner , Dasha Nelidova , Botond Roska
摘要: The present invention relates to an isolated nucleic acid molecule comprising a nucleotide sequence coding for a depolarizing light-gated ion channel functionally linked to a promoter leading to the specific expression of said depolarizing light-gated ion channel in a retinal photoreceptor, or the nucleotide sequence complementary to said nucleotide sequence, for use in treating or ameliorating blindness. The present invention also relates to methods of using such nucleic acid molecules in the treatment of blindness.
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公开(公告)号:US11608365B2
公开(公告)日:2023-03-21
申请号:US17342013
申请日:2021-06-08
摘要: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 300 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in rod photoreceptors of a gene when operatively linked to a nucleic acid sequence coding for said gene.
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公开(公告)号:US11525145B2
公开(公告)日:2022-12-13
申请号:US16346680
申请日:2017-11-01
IPC分类号: C12N15/86 , C12N5/0793 , C12N5/079 , A61K48/00 , C12N15/85
摘要: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 400 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in direction selective retinal ganglion cells of a gene when operatively linked to a nucleic acid sequence coding for said gene.
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公开(公告)号:US10995344B2
公开(公告)日:2021-05-04
申请号:US15780557
申请日:2016-12-01
IPC分类号: C12N15/79 , A61K48/00 , C12N15/86 , C07K14/195 , C07K14/405
摘要: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 400 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in rod photoreceptors of a gene when operatively linked to a nucleic acid sequence coding for said gene.
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