RU2019115117A3 - - Google Patents

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RU2019115117A3
RU2019115117A3 RU2019115117A RU2019115117A RU2019115117A3 RU 2019115117 A3 RU2019115117 A3 RU 2019115117A3 RU 2019115117 A RU2019115117 A RU 2019115117A RU 2019115117 A RU2019115117 A RU 2019115117A RU 2019115117 A3 RU2019115117 A3 RU 2019115117A3
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RU2019115117A
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RU2766353C2 (ru
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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
    • 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
    • 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
    • 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
    • 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
    • C12N5/0621Eye 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified 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
    • 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
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid DNA for vertebrates

Landscapes

  • 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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (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)
RU2019115117A 2016-11-02 2017-11-01 Synp198, промотор для специфической экспрессии генов в ганглионарных клетках сетчатки, избирательных в отношении направления RU2766353C2 (ru)

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
RU2019115117A RU2019115117A (ru) 2020-12-03
RU2019115117A3 true RU2019115117A3 (enExample) 2021-03-23
RU2766353C2 RU2766353C2 (ru) 2022-03-15

Family

ID=57223559

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2019115117A RU2766353C2 (ru) 2016-11-02 2017-11-01 Synp198, промотор для специфической экспрессии генов в ганглионарных клетках сетчатки, избирательных в отношении направления

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
JP2011516091A (ja) 2008-04-18 2011-05-26 ノバルティス・フォルシュングスシュティフトゥング・ツヴァイクニーダーラッスング・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 失明の治療のための新規な治療用ツールおよび方法
US10941417B2 (en) 2015-04-30 2021-03-09 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
US11254934B2 (en) 2015-10-14 2022-02-22 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in retinal endothelial cells
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
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
WO2017093936A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp162, a promoter for the specific expression of genes in rod photoreceptors
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
AU2017354086C1 (en) 2016-11-02 2021-05-06 Friedrich Miescher Institute For Biomedical Research SynP198, a promoter for the specific expression of genes in direction selective retinal ganglion cells
US11371060B2 (en) 2017-02-08 2022-06-28 Friedrich Miescher Institute For Biomedical Research SYNP88, a promoter for the specific expression of genes in retinal ganglion cells
CR20200210A (es) 2017-11-15 2020-09-23 Friedrich Miescher Institute For Biomedical Res Promotor especìfico de las cèlulas del epitelio pigmentario retinal en primates
BR112020010928A2 (pt) 2017-11-30 2020-11-24 Friedrich Miescher Institute For Biomedical Research synpiii, um promotor para a expressão de gene específica em epitélio de pigmento retinal
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)
WO2022178410A1 (en) * 2021-02-22 2022-08-25 The Regents Of The University Of Colorado, A Body Corporate Viral vector-based gene therapy for ocular conditions

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
JP2011516091A (ja) 2008-04-18 2011-05-26 ノバルティス・フォルシュングスシュティフトゥング・ツヴァイクニーダーラッスング・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 失明の治療のための新規な治療用ツールおよび方法
NZ591391A (en) * 2008-10-22 2012-10-26 Quark Pharmaceuticals Inc Non-invasive methods for treating eye disorders using a topical oligonucleotide composition
EP2776459A1 (en) * 2011-11-08 2014-09-17 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Rod cell-specific promoter
EP2888368A1 (en) 2012-08-27 2015-07-01 Friedrich Miescher Institute for Biomedical Research Retinal off circuit-specific promoter
EP3517135B1 (en) * 2013-05-16 2025-11-19 Beacon Therapeutics (USA), Inc. Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
WO2014199299A1 (en) 2013-06-11 2014-12-18 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
US10179917B2 (en) 2014-02-11 2019-01-15 Friedrich Miescher Institute For Biomedical Research Muller 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
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
US11254934B2 (en) 2015-10-14 2022-02-22 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in retinal endothelial cells
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
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
WO2017093936A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp162, a promoter for the specific expression of genes in rod photoreceptors
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
US11390866B2 (en) 2015-12-04 2022-07-19 Sorbonne Université 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
AU2017354086C1 (en) 2016-11-02 2021-05-06 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
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
US11371060B2 (en) 2017-02-08 2022-06-28 Friedrich Miescher Institute For Biomedical Research SYNP88, a promoter for the specific expression of genes in retinal ganglion cells
CR20200210A (es) 2017-11-15 2020-09-23 Friedrich Miescher Institute For Biomedical Res Promotor especìfico de las cèlulas del epitelio pigmentario retinal en primates
BR112020010928A2 (pt) 2017-11-30 2020-11-24 Friedrich Miescher Institute For Biomedical Research synpiii, um promotor para a expressão de gene específica em epitélio de pigmento retinal
KR20200091435A (ko) 2017-11-30 2020-07-30 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 영장류 망막 색소 상피 세포-특이적 프로모터 SynP61
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
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)
WO2020084540A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp78 (proa27), 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
EP3870238A1 (en) 2018-10-25 2021-09-01 Friedrich Miescher Institute for Biomedical Research Synp17 (prob1), 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)
US20220090062A1 (en) 2019-01-24 2022-03-24 Friedrich Miescher Institute For Biomedical Research Synp66 (proa21), a promoter for the specific expression of genes in retinal ganglion cells
EP3914721A1 (en) 2019-01-24 2021-12-01 Friedrich Miescher Institute for Biomedical Research Synp35 (proc8), 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
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
WO2020174368A1 (en) 2019-02-25 2020-09-03 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

Also Published As

Publication number Publication date
AU2017354086C1 (en) 2021-05-06
CA3042197A1 (en) 2018-05-11
JP7023279B2 (ja) 2022-02-21
IL266115A (en) 2019-06-30
RU2019115117A (ru) 2020-12-03
US11525145B2 (en) 2022-12-13
EP3534966B1 (en) 2024-06-19
CN110072557B (zh) 2022-11-11
EP3534966A1 (en) 2019-09-11
AU2017354086B2 (en) 2021-01-28
KR20190077471A (ko) 2019-07-03
CN110072557A (zh) 2019-07-30
JP2019537437A (ja) 2019-12-26
US20190276847A1 (en) 2019-09-12
BR112019008488A2 (pt) 2019-07-09
AU2017354086A1 (en) 2019-05-09
WO2018083607A1 (en) 2018-05-11
RU2766353C2 (ru) 2022-03-15

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