AU2017354086C1 - 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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AU2017354086C1
AU2017354086C1 AU2017354086A AU2017354086A AU2017354086C1 AU 2017354086 C1 AU2017354086 C1 AU 2017354086C1 AU 2017354086 A AU2017354086 A AU 2017354086A AU 2017354086 A AU2017354086 A AU 2017354086A AU 2017354086 C1 AU2017354086 C1 AU 2017354086C1
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cell
nucleic acid
gene
sequence
acid sequence
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AU2017354086B2 (en
AU2017354086A1 (en
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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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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0618Cells of the nervous system
    • C12N5/0619Neurons
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0618Cells of the nervous system
    • C12N5/0621Eye cells, e.g. cornea, iris pigmented cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8527Vectors 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
    • C12N2015/859Animal models comprising reporter system for screening tests
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00Genetically modified cells
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    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/022Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid 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)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Plant Pathology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (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)
AU2017354086A 2016-11-02 2017-11-01 SynP198, a promoter for the specific expression of genes in direction selective retinal ganglion cells Ceased AU2017354086C1 (en)

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)

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AU2017354086A1 AU2017354086A1 (en) 2019-05-09
AU2017354086B2 AU2017354086B2 (en) 2021-01-28
AU2017354086C1 true AU2017354086C1 (en) 2021-05-06

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AU2017354086A Ceased AU2017354086C1 (en) 2016-11-02 2017-11-01 SynP198, a promoter for the specific expression of genes in direction selective retinal ganglion cells

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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=)

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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

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JP7023279B2 (ja) 2022-02-21
KR20190077471A (ko) 2019-07-03
JP2019537437A (ja) 2019-12-26
CN110072557B (zh) 2022-11-11
EP3534966B1 (en) 2024-06-19
US20190276847A1 (en) 2019-09-12
BR112019008488A2 (pt) 2019-07-09
US11525145B2 (en) 2022-12-13
CN110072557A (zh) 2019-07-30
RU2019115117A3 (https=) 2021-03-23
AU2017354086B2 (en) 2021-01-28
WO2018083607A1 (en) 2018-05-11
IL266115A (en) 2019-06-30
CA3042197A1 (en) 2018-05-11
RU2019115117A (ru) 2020-12-03
AU2017354086A1 (en) 2019-05-09
EP3534966A1 (en) 2019-09-11
RU2766353C2 (ru) 2022-03-15

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