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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    • 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
    • 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
    • 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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    • 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
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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
    • C12N15/8509Vectors 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
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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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  • Plant Pathology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • 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)
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
EP16196818.5 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 (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
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
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
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
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
WO2017093935A1 (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
CA3042197A1 (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
CN110392582A (zh) 2017-02-08 2019-10-29 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SynP88
WO2019097454A1 (en) 2017-11-15 2019-05-23 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
WO2019106035A1 (en) 2017-11-30 2019-06-06 Friedrich Miescher Institute For Biomedical Research Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium
EP3870242A1 (en) * 2018-10-25 2021-09-01 Friedrich Miescher Institute for Biomedical Research Synp151 (proc29), a promoter for the specific expression of genes in retinal ganglion cells
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

Family Cites Families (38)

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Publication number Priority date Publication date Assignee Title
EP2019588A4 (en) * 2006-05-04 2010-11-24 Univ Wayne State RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN
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
DK2350279T3 (en) * 2008-10-22 2016-02-15 Quark Pharmaceuticals Inc METHODS OF TREATING EYE DISEASES
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
US9724429B2 (en) * 2013-05-16 2017-08-08 Applied Genetic Technologies Corporation 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
WO2015121793A1 (en) 2014-02-11 2015-08-20 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
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
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
WO2017093935A1 (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
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
AU2016363830C1 (en) 2015-12-04 2022-03-24 Centre National De La Recherche Scientifique 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
CA3042197A1 (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
CN110072559A (zh) 2016-12-01 2019-07-30 弗里德里克·米谢尔生物医学研究所 用于使基因在中间神经元中特异性表达的启动子SynP107
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
CN110392582A (zh) 2017-02-08 2019-10-29 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SynP88
WO2019097454A1 (en) 2017-11-15 2019-05-23 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
BR112020010775A2 (pt) 2017-11-30 2020-11-24 Friedrich Miescher Institute For Biomedical Research synp61, promotor específico à célula de epitélio de pigmento retiniano de primata
WO2019106035A1 (en) 2017-11-30 2019-06-06 Friedrich Miescher Institute For Biomedical Research Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium
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
EP3870242A1 (en) 2018-10-25 2021-09-01 Friedrich Miescher Institute for Biomedical Research Synp151 (proc29), 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
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
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
US20210355505A1 (en) 2018-10-25 2021-11-18 Friedrich Miescher Institute For Biomedical Research Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells
WO2020152624A1 (en) 2019-01-24 2020-07-30 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
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
JP2022521025A (ja) 2019-02-25 2022-04-04 ノバルティス アーゲー Biettiクリスタリン網膜症を治療するための組成物及び方法
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
EP3534966A1 (en) 2019-09-11
CN110072557B (zh) 2022-11-11
BR112019008488A2 (pt) 2019-07-09
JP2019537437A (ja) 2019-12-26
KR20190077471A (ko) 2019-07-03
RU2766353C2 (ru) 2022-03-15
RU2019115117A3 (enExample) 2021-03-23
AU2017354086A1 (en) 2019-05-09
AU2017354086B2 (en) 2021-01-28
JP7023279B2 (ja) 2022-02-21
IL266115A (en) 2019-06-30
RU2019115117A (ru) 2020-12-03
US11525145B2 (en) 2022-12-13
WO2018083607A1 (en) 2018-05-11
AU2017354086C1 (en) 2021-05-06
CN110072557A (zh) 2019-07-30
EP3534966B1 (en) 2024-06-19
US20190276847A1 (en) 2019-09-12

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