CA3082080A1 - Primate retinal pigment epithelium cell-specific promoter - Google Patents

Primate retinal pigment epithelium cell-specific promoter Download PDF

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Publication number
CA3082080A1
CA3082080A1 CA3082080A CA3082080A CA3082080A1 CA 3082080 A1 CA3082080 A1 CA 3082080A1 CA 3082080 A CA3082080 A CA 3082080A CA 3082080 A CA3082080 A CA 3082080A CA 3082080 A1 CA3082080 A1 CA 3082080A1
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Canada
Prior art keywords
nucleic acid
acid sequence
sequence
cells
acid molecule
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Pending
Application number
CA3082080A
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English (en)
French (fr)
Inventor
Josephine JUETTNER
Jacek Krol
Botond Roska
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
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 CA3082080A1 publication Critical patent/CA3082080A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0393—Animal model comprising a 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Environmental Sciences (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CA3082080A 2017-11-15 2018-11-15 Primate retinal pigment epithelium cell-specific promoter Pending CA3082080A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP17201828 2017-11-15
EP17201828.5 2017-11-15
PCT/IB2018/059015 WO2019097454A1 (en) 2017-11-15 2018-11-15 Primate retinal pigment epithelium cell-specific promoter

Publications (1)

Publication Number Publication Date
CA3082080A1 true CA3082080A1 (en) 2019-05-23

Family

ID=60331462

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3082080A Pending CA3082080A1 (en) 2017-11-15 2018-11-15 Primate retinal pigment epithelium cell-specific promoter

Country Status (14)

Country Link
US (1) US11739349B2 (enExample)
EP (1) EP3709798B1 (enExample)
JP (2) JP7348176B2 (enExample)
KR (1) KR20200084028A (enExample)
CN (1) CN111726985B (enExample)
AU (1) AU2018369975B2 (enExample)
BR (1) BR112020009579A2 (enExample)
CA (1) CA3082080A1 (enExample)
CR (1) CR20200210A (enExample)
IL (1) IL274460A (enExample)
MX (1) MX2020005033A (enExample)
PH (1) PH12020550757A1 (enExample)
SG (1) SG11202004205YA (enExample)
WO (1) WO2019097454A1 (enExample)

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ES2747433T3 (es) 2015-04-30 2020-03-10 Friedrich Miescher Institute For Biomedical Res Promotor para la expresión específica de genes en células de Müller
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
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
CN108430519B (zh) 2015-12-03 2022-04-15 弗里德里克·米谢尔生物医学研究所 SynP159，用于基因在视杆光感受器中特异性表达的启动子
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
JP7075341B2 (ja) 2015-12-03 2022-05-25 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 ＳｙｎＰ１６２、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
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
KR20190077471A (ko) 2016-11-02 2019-07-03 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 방향 선택적인 망막 신경절 세포에서의 유전자의 특이적 발현을 위한 프로모터, synp198
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
AU2018369975B2 (en) 2017-11-15 2022-05-19 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
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
WO2019106027A1 (en) * 2017-11-30 2019-06-06 Friedrich Miescher Institute For Biomedical Research SynP61, A PRIMATE RETINAL PIGMENT EPITHELIUM CELL-SPECIFIC PROMOTER
CN114045264B (zh) * 2021-11-16 2024-05-14 珠海中科先进技术研究院有限公司 一种获取驱动元件的方法
WO2024033837A1 (en) * 2022-08-11 2024-02-15 Institute Of Molecular And Clinical Ophthalmology Basel (Iob) Human cone photoreceptor optogenetic constructs

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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
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
JP7075341B2 (ja) 2015-12-03 2022-05-25 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 ＳｙｎＰ１６２、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
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
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
CN108430519B (zh) 2015-12-03 2022-04-15 弗里德里克·米谢尔生物医学研究所 SynP159，用于基因在视杆光感受器中特异性表达的启动子
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US20190209708A1 (en) 2016-05-17 2019-07-11 Friedrich Miescher Institute For Biomedical Research Novel therapeutic tools and methods for treating blindness
KR20190077471A (ko) 2016-11-02 2019-07-03 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 방향 선택적인 망막 신경절 세포에서의 유전자의 특이적 발현을 위한 프로모터, synp198
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
CN110072558A (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SynPI
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
AU2018369975B2 (en) 2017-11-15 2022-05-19 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
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
WO2019106027A1 (en) 2017-11-30 2019-06-06 Friedrich Miescher Institute For Biomedical Research SynP61, A PRIMATE RETINAL PIGMENT EPITHELIUM CELL-SPECIFIC PROMOTER
JP2022505516A (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ＳｙｎＰ１７（ＰｒｏＢ１）
US20210388386A1 (en) 2018-10-25 2021-12-16 Friedrich Miescher Institute For Biomedical Research Synp57 (proa14), a promoter for the specific expression of genes in photoreceptors
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
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
JP2022512780A (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ＳｙｎＰ１９４（ＰｒｏＢ１５）
WO2020084538A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp27 (prob12), a promoter for the specific expression of genes in protoplasmic astrocytes
EP3914299A1 (en) 2019-01-24 2021-12-01 Friedrich Miescher Institute for Biomedical Research Synp5 (proa9), 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
US20220119807A1 (en) 2019-01-24 2022-04-21 Friedrich Miescher Institute For Biomedical Research Synp35 (proc8), a promoter for the specific expression of genes in retinal ganglion cells
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
WO2020174369A2 (en) 2019-02-25 2020-09-03 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy

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US20210054408A1 (en) 2021-02-25
RU2020119176A3 (enExample) 2022-01-26
JP2023075139A (ja) 2023-05-30
WO2019097454A1 (en) 2019-05-23
EP3709798A1 (en) 2020-09-23
MX2020005033A (es) 2020-10-28
SG11202004205YA (en) 2020-06-29
AU2018369975B2 (en) 2022-05-19
KR20200084028A (ko) 2020-07-09
AU2018369975A1 (en) 2020-05-28
JP2021502813A (ja) 2021-02-04
BR112020009579A2 (pt) 2020-10-13
CN111726985A (zh) 2020-09-29
JP7348176B2 (ja) 2023-09-20
CR20200210A (es) 2020-09-23
IL274460A (en) 2020-06-30
US11739349B2 (en) 2023-08-29
PH12020550757A1 (en) 2021-05-10
RU2020119176A (ru) 2021-12-16
CN111726985B (zh) 2022-06-10
EP3709798B1 (en) 2024-04-17

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