CA3042197A1 - Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells - Google Patents

Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells Download PDF

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Publication number
CA3042197A1
CA3042197A1 CA3042197A CA3042197A CA3042197A1 CA 3042197 A1 CA3042197 A1 CA 3042197A1 CA 3042197 A CA3042197 A CA 3042197A CA 3042197 A CA3042197 A CA 3042197A CA 3042197 A1 CA3042197 A1 CA 3042197A1
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Prior art keywords
nucleic acid
gene
sequence
cell
acid sequence
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Abandoned
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CA3042197A
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English (en)
French (fr)
Inventor
Dominik Hartl
Josephine JUETTNER
Arnaud KREBS
Botond Roska
Dirk Schuebeler
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Novartis Forschungsstiftung Zweigniederlassung Friedrich Miescher Institute for Biomedical Research
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Novartis Forschungsstiftung Zweigniederlassung Friedrich Miescher Institute for Biomedical Research
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Publication of CA3042197A1 publication Critical patent/CA3042197A1/en
Abandoned legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0619—Neurons
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0621—Eye cells, e.g. cornea, iris pigmented cells
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8527—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
    • C12N2015/859—Animal models comprising reporter system for screening tests
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Cell Biology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3042197A 2016-11-02 2017-11-01 Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells Abandoned CA3042197A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16196818 2016-11-02
EP16196818.5 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 (1)

Publication Number Publication Date
CA3042197A1 true CA3042197A1 (en) 2018-05-11

Family

ID=57223559

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3042197A Abandoned CA3042197A1 (en) 2016-11-02 2017-11-01 Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells

Country Status (11)

Country Link
US (1) US11525145B2 (enExample)
EP (1) EP3534966B1 (enExample)
JP (1) JP7023279B2 (enExample)
KR (1) KR20190077471A (enExample)
CN (1) CN110072557B (enExample)
AU (1) AU2017354086C1 (enExample)
BR (1) BR112019008488A2 (enExample)
CA (1) CA3042197A1 (enExample)
IL (1) IL266115A (enExample)
RU (1) RU2766353C2 (enExample)
WO (1) WO2018083607A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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.
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
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
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
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)
JP7895637B2 (ja) * 2021-02-22 2026-07-28 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
US8470790B2 (en) * 2006-05-04 2013-06-25 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
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
ES2563984T3 (es) * 2008-10-22 2016-03-17 Quark Pharmaceuticals, Inc. Métodos para el tratamiento de trastornos oculares
WO2013068413A1 (en) * 2011-11-08 2013-05-16 Novartis Forschungsstiftung, Zweigniederlassung, Friedrich Miescher Institute For Biomedical Research Rod cell-specific promoter
US9579399B2 (en) 2012-08-27 2017-02-28 Friedrich Miescher Institute For Biomedical Research Retinal OFF circuit-specific promoter
EP2996725A4 (en) * 2013-05-16 2016-12-28 Applied Genetic Tech Corp PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS AND METHODS FOR TREATING COLOR BLINDNESS AND OTHER PROBLEMS
US9862970B2 (en) 2013-06-11 2018-01-09 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
WO2015121793A1 (en) 2014-02-11 2015-08-20 Friedrich Miescher Institute For Biomedical Research Müller cell-specific promoter
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
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
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
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
WO2018099974A1 (en) 2016-12-01 2018-06-07 Friedrich Miescher Institute For Biomedical Research Synp107, a promoter for the specific expression of genes in interneurons
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
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
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
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)
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
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
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
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
WO2020152623A1 (en) 2019-01-24 2020-07-30 Friedrich Miescher Institute For Biomedical Research Synp5 (proa9), a promoter for the specific expression of genes in retinal ganglion cells
JP2022517688A (ja) 2019-01-24 2022-03-09 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
US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
EP3931335A2 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy

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Publication number Publication date
US11525145B2 (en) 2022-12-13
BR112019008488A2 (pt) 2019-07-09
US20190276847A1 (en) 2019-09-12
JP2019537437A (ja) 2019-12-26
RU2019115117A (ru) 2020-12-03
CN110072557A (zh) 2019-07-30
WO2018083607A1 (en) 2018-05-11
EP3534966A1 (en) 2019-09-11
KR20190077471A (ko) 2019-07-03
JP7023279B2 (ja) 2022-02-21
AU2017354086C1 (en) 2021-05-06
AU2017354086B2 (en) 2021-01-28
AU2017354086A1 (en) 2019-05-09
RU2019115117A3 (enExample) 2021-03-23
IL266115A (en) 2019-06-30
RU2766353C2 (ru) 2022-03-15
CN110072557B (zh) 2022-11-11
EP3534966B1 (en) 2024-06-19

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