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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JP
Japan
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nucleic acid
cell
isolated nucleic
vector
expression cassette
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JP2019522550A
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Japanese (ja)
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JP7023279B2 (ja
JP2019537437A (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 (https=) 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 (https=)
EP (1) EP3534966B1 (https=)
JP (1) JP7023279B2 (https=)
KR (1) KR20190077471A (https=)
CN (1) CN110072557B (https=)
AU (1) AU2017354086C1 (https=)
BR (1) BR112019008488A2 (https=)
CA (1) CA3042197A1 (https=)
IL (1) IL266115A (https=)
RU (1) RU2766353C2 (https=)
WO (1) WO2018083607A1 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2010011467A (es) 2008-04-18 2011-02-15 Novartis Forschungsstiftung Novedosas herramientas y metodos terapeuticos para tratar la ceguera.
US10941417B2 (en) 2015-04-30 2021-03-09 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in Müller cells
US10857241B2 (en) 2015-09-15 2020-12-08 Friedrich Miescher Institute For Biomedical Research Therapeutical tools and methods for treating blindness by targeting photoreceptors
WO2017064642A1 (en) 2015-10-14 2017-04-20 Fredrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in retinal endothelial cells
EP3383440B1 (en) 2015-12-03 2021-06-30 Friedrich Miescher Institute for Biomedical Research Synp162, a promoter for the specific expression of genes in rod photoreceptors
EP3383439B1 (en) 2015-12-03 2019-09-25 Friedrich Miescher Institute for Biomedical Research Synp161, a promoter for the specific expression of genes in rod photoreceptors
WO2017093934A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp160, a promoter for the specific expression of genes in rod photoreceptors
ES2909444T3 (es) 2015-12-03 2022-05-06 Friedrich Miescher Institute For Biomedical Res Synp159, un promotor para la expresión específica de genes en los fotorreceptores de los bastones
RU2766353C2 (ru) 2016-11-02 2022-03-15 Фридрих Мишер Инститьют Фор Байомедикал Рисерч Synp198, промотор для специфической экспрессии генов в ганглионарных клетках сетчатки, избирательных в отношении направления
WO2018146588A1 (en) 2017-02-08 2018-08-16 Friedrich Miescher Institute For Biomedical Research Synp88, a promoter for the specific expression of genes in retinal ganglion cells
MX2020005033A (es) 2017-11-15 2020-10-28 Friedrich Miescher Institute For Biomedical Res Promotor especifico de las celulas del epitelio pigmentario retinal en primates.
SG11202005024QA (en) 2017-11-30 2020-06-29 Friedrich Miescher Institute For Biomedical Res Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium
JP2022505517A (ja) * 2018-10-25 2022-01-14 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SynP151(ProC29)
JP2024507860A (ja) * 2021-02-22 2024-02-21 ザ・リージエンツ・オブ・ザ・ユニバーシテイ・オブ・コロラド、ア・ボデイー・コーポレイト 眼病態に対するウイルスベクターベースの遺伝子療法

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101484005A (zh) * 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
EP1891976A1 (en) 2006-08-23 2008-02-27 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute for Biomedical Research Use of light sensitive genes
MX2010011467A (es) 2008-04-18 2011-02-15 Novartis Forschungsstiftung Novedosas herramientas y metodos terapeuticos para tratar la ceguera.
CA2962219C (en) * 2008-10-22 2020-08-25 Quark Pharmaceuticals, Inc. Methods for treating eye disorders
EP2776459A1 (en) * 2011-11-08 2014-09-17 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Rod cell-specific promoter
WO2014033095A1 (en) 2012-08-27 2014-03-06 Friedrich Miescher Institute For Biomedical Research Retinal off circuit-specific promoter
CN105764531A (zh) * 2013-05-16 2016-07-13 应用遗传科技公司 用于治疗色盲和其它疾病的启动子、表达盒、载体、药盒和方法
EP3007730A1 (en) 2013-06-11 2016-04-20 Friedrich Miescher Institute for Biomedical Research Retinal on bipolar cells-specific artificial promoter
EP3104895A1 (en) 2014-02-10 2016-12-21 Friedrich Miescher Institute for Biomedical Research Aii retinal amacrine cell-specific promoter
EP3105334B1 (en) 2014-02-11 2019-07-03 Friedrich Miescher Institute for Biomedical Research Müller cell-specific promoter
US10941417B2 (en) 2015-04-30 2021-03-09 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in Müller cells
US10857241B2 (en) 2015-09-15 2020-12-08 Friedrich Miescher Institute For Biomedical Research Therapeutical tools and methods for treating blindness by targeting photoreceptors
WO2017064642A1 (en) 2015-10-14 2017-04-20 Fredrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in retinal endothelial cells
EP3383439B1 (en) 2015-12-03 2019-09-25 Friedrich Miescher Institute for Biomedical Research Synp161, a promoter for the specific expression of genes in rod photoreceptors
ES2909444T3 (es) 2015-12-03 2022-05-06 Friedrich Miescher Institute For Biomedical Res Synp159, un promotor para la expresión específica de genes en los fotorreceptores de los bastones
WO2017093934A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp160, a promoter for the specific expression of genes in rod photoreceptors
EP3383440B1 (en) 2015-12-03 2021-06-30 Friedrich Miescher Institute for Biomedical Research Synp162, a promoter for the specific expression of genes in rod photoreceptors
WO2017093566A1 (en) 2015-12-04 2017-06-08 Universite Pierre Et Marie Curie (Paris 6) Promoters and uses thereof
US20190209708A1 (en) 2016-05-17 2019-07-11 Friedrich Miescher Institute For Biomedical Research Novel therapeutic tools and methods for treating blindness
RU2766353C2 (ru) 2016-11-02 2022-03-15 Фридрих Мишер Инститьют Фор Байомедикал Рисерч Synp198, промотор для специфической экспрессии генов в ганглионарных клетках сетчатки, избирательных в отношении направления
EP3548093A1 (en) 2016-12-01 2019-10-09 Friedrich Miescher Institute for Biomedical Research Synp107, a promoter for the specific expression of genes in interneurons
US20190376083A1 (en) 2016-12-01 2019-12-12 Friedrich Miescher Institute For Biomedical Research Synp1, a promoter for the specific expression of genes in interneurons
WO2018146588A1 (en) 2017-02-08 2018-08-16 Friedrich Miescher Institute For Biomedical Research Synp88, a promoter for the specific expression of genes in retinal ganglion cells
MX2020005033A (es) 2017-11-15 2020-10-28 Friedrich Miescher Institute For Biomedical Res Promotor especifico de las celulas del epitelio pigmentario retinal en primates.
CN111527102A (zh) 2017-11-30 2020-08-11 弗里德里克·米谢尔生物医学研究所 灵长类动物视网膜色素上皮细胞特异性启动子SynP61
SG11202005024QA (en) 2017-11-30 2020-06-29 Friedrich Miescher Institute For Biomedical Res Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium
JP2022505517A (ja) 2018-10-25 2022-01-14 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SynP151(ProC29)
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
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
EP3870239A1 (en) 2018-10-25 2021-09-01 Friedrich Miescher Institute for Biomedical Research Synp57 (proa14), a promoter for the specific expression of genes in photoreceptors
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
US20210353773A1 (en) 2018-10-25 2021-11-18 Friedrich Miescher Institute For Biomedical Research Synp194 (prob15), a promoter for the specific expression of genes in retinal ganglion cells
US20220119807A1 (en) 2019-01-24 2022-04-21 Friedrich Miescher Institute For Biomedical Research Synp35 (proc8), a promoter for the specific expression of genes in retinal ganglion cells
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
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
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy

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