JP2019537437A5 - - Google Patents

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JP2019537437A5
JP2019537437A5 JP2019522550A JP2019522550A JP2019537437A5 JP 2019537437 A5 JP2019537437 A5 JP 2019537437A5 JP 2019522550 A JP2019522550 A JP 2019522550A JP 2019522550 A JP2019522550 A JP 2019522550A JP 2019537437 A5 JP2019537437 A5 JP 2019537437A5
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Japan
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nucleic acid
cell
isolated nucleic
vector
expression cassette
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JP2019522550A
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JP2019537437A (ja
JP7023279B2 (ja
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Priority claimed from PCT/IB2017/056792 external-priority patent/WO2018083607A1/en
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JP2019522550A 2016-11-02 2017-11-01 SynP198、方向選択性網膜神経節細胞内での遺伝子の特異的発現のためのプロモーター Expired - Fee Related JP7023279B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16196818.5 2016-11-02
EP16196818 2016-11-02
PCT/IB2017/056792 WO2018083607A1 (en) 2016-11-02 2017-11-01 Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells

Publications (3)

Publication Number Publication Date
JP2019537437A JP2019537437A (ja) 2019-12-26
JP2019537437A5 true JP2019537437A5 (cg-RX-API-DMAC7.html) 2020-12-10
JP7023279B2 JP7023279B2 (ja) 2022-02-21

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JP2019522550A Expired - Fee Related JP7023279B2 (ja) 2016-11-02 2017-11-01 SynP198、方向選択性網膜神経節細胞内での遺伝子の特異的発現のためのプロモーター

Country Status (11)

Country Link
US (1) US11525145B2 (cg-RX-API-DMAC7.html)
EP (1) EP3534966B1 (cg-RX-API-DMAC7.html)
JP (1) JP7023279B2 (cg-RX-API-DMAC7.html)
KR (1) KR20190077471A (cg-RX-API-DMAC7.html)
CN (1) CN110072557B (cg-RX-API-DMAC7.html)
AU (1) AU2017354086C1 (cg-RX-API-DMAC7.html)
BR (1) BR112019008488A2 (cg-RX-API-DMAC7.html)
CA (1) CA3042197A1 (cg-RX-API-DMAC7.html)
IL (1) IL266115A (cg-RX-API-DMAC7.html)
RU (1) RU2766353C2 (cg-RX-API-DMAC7.html)
WO (1) WO2018083607A1 (cg-RX-API-DMAC7.html)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20100136550A (ko) 2008-04-18 2010-12-28 노파르티스 포르슝스티프퉁 쯔바이크니덜라쑹 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 실명을 치료하기 위한 신규의 치료 도구 및 방법
EP3289090B1 (en) 2015-04-30 2019-06-26 Friedrich Miescher Institute for Biomedical Research Promoter for the specific expression of genes in müller cells
ES2926677T3 (es) 2015-09-15 2022-10-27 Friedrich Miescher Institute For Biomedical Res Herramientas y métodos terapéuticos novedosos para tratar la ceguera mediante el direccionamiento a los fotorreceptores
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.
ES2886664T3 (es) 2015-12-03 2021-12-20 Friedrich Miescher Institute For Biomedical Res SynP162, un promotor para la expresión específica de genes en fotorreceptores de bastón
CN108430519B (zh) 2015-12-03 2022-04-15 弗里德里克·米谢尔生物医学研究所 SynP159,用于基因在视杆光感受器中特异性表达的启动子
ES2761328T3 (es) 2015-12-03 2020-05-19 Friedrich Miescher Institute For Biomedical Res SynP161, un promotor para la expresión específica de genes en fotorreceptores de bastón
CN108474001B (zh) 2015-12-03 2022-04-08 弗里德里克·米谢尔生物医学研究所 SynP160,用于基因在视杆光感受器中特异性表达的启动子
CA3042197A1 (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
CA3082080A1 (en) 2017-11-15 2019-05-23 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
EP3717651A1 (en) 2017-11-30 2020-10-07 Friedrich Miescher Institute for Biomedical Research Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium
WO2020084541A1 (en) * 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp151 (proc29), a promoter for the specific expression of genes in retinal ganglion cells
CN117157091A (zh) * 2021-02-22 2023-12-01 科罗拉多大学董事会法人团体 基于病毒载体的眼部病症的基因治疗

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3437473A1 (en) * 2006-05-04 2019-02-06 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
EP1891976A1 (en) 2006-08-23 2008-02-27 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute for Biomedical Research Use of light sensitive genes
KR20100136550A (ko) 2008-04-18 2010-12-28 노파르티스 포르슝스티프퉁 쯔바이크니덜라쑹 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 실명을 치료하기 위한 신규의 치료 도구 및 방법
CN104857526B (zh) * 2008-10-22 2019-03-05 夸克制药公司 治疗眼部疾病的方法
US20140287510A1 (en) * 2011-11-08 2014-09-25 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute Rod cell-specific promoter
US9579399B2 (en) 2012-08-27 2017-02-28 Friedrich Miescher Institute For Biomedical Research Retinal OFF circuit-specific promoter
AU2014265799B2 (en) * 2013-05-16 2019-10-31 Applied Genetic Technologies Corporation Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
EP3007730A1 (en) 2013-06-11 2016-04-20 Friedrich Miescher Institute for Biomedical Research Retinal on bipolar cells-specific artificial promoter
WO2015118507A1 (en) 2014-02-10 2015-08-13 Friedrich Miescher Institute For Biomedical Research Aii retinal amacrine cell-specific promoter
US10179917B2 (en) 2014-02-11 2019-01-15 Friedrich Miescher Institute For Biomedical Research Muller cell-specific promoter
EP3289090B1 (en) 2015-04-30 2019-06-26 Friedrich Miescher Institute for Biomedical Research Promoter for the specific expression of genes in müller cells
ES2926677T3 (es) 2015-09-15 2022-10-27 Friedrich Miescher Institute For Biomedical Res Herramientas y métodos terapéuticos novedosos para tratar la ceguera mediante el direccionamiento a los fotorreceptores
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.
ES2761328T3 (es) 2015-12-03 2020-05-19 Friedrich Miescher Institute For Biomedical Res SynP161, un promotor para la expresión específica de genes en fotorreceptores de bastón
CN108474001B (zh) 2015-12-03 2022-04-08 弗里德里克·米谢尔生物医学研究所 SynP160,用于基因在视杆光感受器中特异性表达的启动子
ES2886664T3 (es) 2015-12-03 2021-12-20 Friedrich Miescher Institute For Biomedical Res SynP162, un promotor para la expresión específica de genes en fotorreceptores de bastón
CN108430519B (zh) 2015-12-03 2022-04-15 弗里德里克·米谢尔生物医学研究所 SynP159,用于基因在视杆光感受器中特异性表达的启动子
CA3004807C (en) 2015-12-04 2022-02-22 Universite Pierre Et Marie Curie (Paris 6) Promoters and uses thereof
WO2017199156A1 (en) 2016-05-17 2017-11-23 Friedrich Miescher Institute For Biomedical Research Novel therapeutic tools and methods for treating blindness
CA3042197A1 (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
JP2020501538A (ja) 2016-12-01 2020-01-23 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 インターニューロン中の遺伝子の特異的発現のためのプロモーターsynpi
JP7071361B2 (ja) 2016-12-01 2022-05-18 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 インターニューロン中の遺伝子の特異的発現のためのプロモーターsynp107
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
CA3082080A1 (en) 2017-11-15 2019-05-23 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
EP3717651A1 (en) 2017-11-30 2020-10-07 Friedrich Miescher Institute for Biomedical Research Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium
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
EP3870240A1 (en) 2018-10-25 2021-09-01 Friedrich Miescher Institute for Biomedical Research Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells
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
EP3870709A1 (en) 2018-10-25 2021-09-01 Friedrich Miescher Institute for Biomedical Research Synp27 (prob12), a promoter for the specific expression of genes in protoplasmic astrocytes
WO2020084539A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp57 (proa14), a promoter for the specific expression of genes in photoreceptors
WO2020084541A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp151 (proc29), a promoter for the specific expression of genes in retinal ganglion cells
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)
US20220119841A1 (en) 2019-01-24 2022-04-21 Friedrich Miescher Instit for Biomedical Research Synp5 (proa9), a promoter for the specific expression of genes in retinal ganglion cells
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
CN113316643A (zh) 2019-01-24 2021-08-27 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SynP35(ProC8)
EP3914300A1 (en) 2019-01-24 2021-12-01 Friedrich Miescher Institute for Biomedical Research Synp66 (proa21), a promoter for the specific expression of genes in retinal ganglion cells
PH12021552038A1 (en) 2019-02-25 2023-01-04 Friedrich Miescher Institute For Biomedical Res Compositions and methods to treat bietti crystalline dystrophy
JP2022520875A (ja) 2019-02-25 2022-04-01 ノバルティス アーゲー Biettiクリスタリン網膜症を治療するための組成物及び方法

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