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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AU2017354086A1 (en
AU2017354086B2 (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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    • 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
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/86Viral vectors
    • 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
    • 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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    • 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)
  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Neurosurgery (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 (OSRAM)
EP (1) EP3534966B1 (OSRAM)
JP (1) JP7023279B2 (OSRAM)
KR (1) KR20190077471A (OSRAM)
CN (1) CN110072557B (OSRAM)
AU (1) AU2017354086C1 (OSRAM)
BR (1) BR112019008488A2 (OSRAM)
CA (1) CA3042197A1 (OSRAM)
IL (1) IL266115A (OSRAM)
RU (1) RU2766353C2 (OSRAM)
WO (1) WO2018083607A1 (OSRAM)

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EP3362101B1 (en) 2015-10-14 2021-05-19 Friedrich Miescher Institute for Biomedical Research Promoter for the specific expression of genes in retinal endothelial cells
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
CA3006952A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp161, a promoter for the specific expression of genes in rod photoreceptors
ES2900486T3 (es) 2015-12-03 2022-03-17 Friedrich Miescher Institute For Biomedical Res SynP160, un promotor para la expresión específica de genes en los fotorreceptores de los bastones
WO2017093931A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp159, a promoter for the specific expression of genes in rod photoreceptors
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
CA3082080A1 (en) 2017-11-15 2019-05-23 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
CN111542611A (zh) 2017-11-30 2020-08-14 弗里德里克·米谢尔生物医学研究所 用于使基因在视网膜色素上皮中特异性表达的启动子SynPIII
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KR20230148207A (ko) * 2021-02-22 2023-10-24 더 리전츠 오브 더 유니버시티 오브 콜로라도, 어 바디 코퍼레이트 안구 병태에 대한 바이러스 벡터-기반 유전자 요법

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

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