JP7112414B2 - 網膜神経節細胞中の遺伝子の特異的発現のためのプロモーターsynp88 - Google Patents

網膜神経節細胞中の遺伝子の特異的発現のためのプロモーターsynp88 Download PDF

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JP7112414B2
JP7112414B2 JP2019542436A JP2019542436A JP7112414B2 JP 7112414 B2 JP7112414 B2 JP 7112414B2 JP 2019542436 A JP2019542436 A JP 2019542436A JP 2019542436 A JP2019542436 A JP 2019542436A JP 7112414 B2 JP7112414 B2 JP 7112414B2
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ロスカ,ボトンド
ユエットナー,ジョゼフィーヌ
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フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

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JP2019542436A 2017-02-08 2018-02-05 網膜神経節細胞中の遺伝子の特異的発現のためのプロモーターsynp88 Expired - Fee Related JP7112414B2 (ja)

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EP17155193 2017-02-08
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PCT/IB2018/050696 WO2018146588A1 (en) 2017-02-08 2018-02-05 Synp88, a promoter for the specific expression of genes in retinal ganglion cells

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EP2679246A3 (en) 2008-04-18 2014-02-26 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Novel therapeutical tools and methods for treating blindness
WO2016174624A1 (en) 2015-04-30 2016-11-03 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in müller cells
WO2017046084A1 (en) 2015-09-15 2017-03-23 Friedrich Miescher Institute For Biomedical Research Novel therapeutical tools and methods for treating blindness by targeting photoreceptors
ES2881782T3 (es) 2015-10-14 2021-11-30 Friedrich Miescher Institute For Biomedical Res Promotor para la expresión específica de genes en células endoteliales retinianas.
US11059871B2 (en) 2015-12-03 2021-07-13 Friedrich Miescher Institute For Biomedical Research SYNP162, a promoter for the specific expression of genes in rod photoreceptors
US10898586B2 (en) 2015-12-03 2021-01-26 Friedrich Miescher Institute For Biomedical Research SynP161, a promoter for the specific expression of genes in rod photoreceptors
JP7058220B2 (ja) 2015-12-03 2022-04-21 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ SynP159、桿体光受容器における遺伝子の特異的発現のためのプロモーター
US10994026B2 (en) 2015-12-03 2021-05-04 Friedrich Miescher Institute For Biomedical Research SynP160, a promoter for the specific expression of genes in rod photoreceptors
WO2018083607A1 (en) 2016-11-02 2018-05-11 Friedrich Miescher Institute For Biomedical Research Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells
JP7112414B2 (ja) * 2017-02-08 2022-08-03 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ 網膜神経節細胞中の遺伝子の特異的発現のためのプロモーターsynp88
WO2019097454A1 (en) 2017-11-15 2019-05-23 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
US11591615B2 (en) 2017-11-30 2023-02-28 Friedrich Miescher Institute For Biomedical Research SynPIII, a promoter for the specific expression of genes in retinal pigment epithelium
CN110857440B (zh) * 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
CN112912508A (zh) * 2018-10-25 2021-06-04 弗里德里克·米谢尔生物医学研究所 用于使基因在视网膜神经节细胞中特异性表达的启动子SynP194(ProB15)
US20210388385A1 (en) * 2018-10-25 2021-12-16 Friedrich Miescher Institute For Biomedical Research Synp27 (prob12), a promoter for the specific expression of genes in protoplasmic astrocytes
IL300277A (en) * 2020-08-05 2023-04-01 Hangzhou Jiayin Biotech Ltd Constructive nucleic acid and its uses for the treatment of spinal muscular atrophy
AR127674A1 (es) 2021-10-18 2024-02-21 Regeneron Pharma Células de mamífero que comprenden genes cas9 integrados para producir sitios de integración estables, y células de mamífero que comprenden sitios de integración estables y otros sitios
JP2024537896A (ja) 2021-10-18 2024-10-16 リジェネロン・ファーマシューティカルズ・インコーポレイテッド アデノウイルス随伴ウイルスポリヌクレオチドを含む真核細胞
CN120098992A (zh) * 2023-12-05 2025-06-06 云舟生物科技(广州)股份有限公司 核酸分子及其作为特异启动子的应用

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JP7058220B2 (ja) 2015-12-03 2022-04-21 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ SynP159、桿体光受容器における遺伝子の特異的発現のためのプロモーター
US10994026B2 (en) 2015-12-03 2021-05-04 Friedrich Miescher Institute For Biomedical Research SynP160, a promoter for the specific expression of genes in rod photoreceptors
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JP6966442B2 (ja) 2015-12-04 2021-11-17 ソルボンヌ・ユニヴェルシテSorbonne Universite プロモーター及びその使用
EP3458476A1 (en) 2016-05-17 2019-03-27 Friedrich Miescher Institute for Biomedical Research Novel therapeutic tools and methods for treating blindness
WO2018083607A1 (en) 2016-11-02 2018-05-11 Friedrich Miescher Institute For Biomedical Research Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells
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CR20200235A (es) 2017-11-30 2020-10-19 Friedrich Miescher Institute For Biomedical Res Synp61, un promotor específico de células del epitelio pigmentario retiniano de primates
US11591615B2 (en) 2017-11-30 2023-02-28 Friedrich Miescher Institute For Biomedical Research SynPIII, a promoter for the specific expression of genes in retinal pigment epithelium
CN112930200A (zh) 2018-10-25 2021-06-08 弗里德里克·米谢尔生物医学研究所 用于视网膜神经节细胞中特异表达基因的启动子SynP151(ProC29)
US20210388385A1 (en) 2018-10-25 2021-12-16 Friedrich Miescher Institute For Biomedical Research Synp27 (prob12), a promoter for the specific expression of genes in protoplasmic astrocytes
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WO2020084537A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp17 (prob1), a promoter for the specific expression of genes in retinal ganglion cells
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JP2022512784A (ja) 2018-10-25 2022-02-07 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ 網膜神経節細胞中の遺伝子の特異的発現のためのプロモーターSynP78(ProA27)
WO2020152626A1 (en) 2019-01-24 2020-07-30 Friedrich Miescher Institute For Biomedical Research Synp166 (proa36), a promoter for the specific expression of genes in photoreceptors
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US20200277626A1 (en) 2020-09-03
CN110392582A (zh) 2019-10-29
US20220267800A1 (en) 2022-08-25
EP3579884A1 (en) 2019-12-18
WO2018146588A1 (en) 2018-08-16
WO2018146588A8 (en) 2019-09-26
US11371060B2 (en) 2022-06-28
JP2020506710A (ja) 2020-03-05

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