CN111527102A - 灵长类动物视网膜色素上皮细胞特异性启动子SynP61 - Google Patents

灵长类动物视网膜色素上皮细胞特异性启动子SynP61 Download PDF

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Publication number
CN111527102A
CN111527102A CN201880084798.9A CN201880084798A CN111527102A CN 111527102 A CN111527102 A CN 111527102A CN 201880084798 A CN201880084798 A CN 201880084798A CN 111527102 A CN111527102 A CN 111527102A
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nucleic acid
acid molecule
isolated nucleic
sequence
acid sequence
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Chinese (zh)
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J·于特纳
J·科洛尔
B·罗斯卡
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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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    • 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
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201880084798.9A 2017-11-30 2018-11-28 灵长类动物视网膜色素上皮细胞特异性启动子SynP61 Pending CN111527102A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP17204746 2017-11-30
EP17204746.6 2017-11-30
PCT/EP2018/082872 WO2019106027A1 (en) 2017-11-30 2018-11-28 SynP61, A PRIMATE RETINAL PIGMENT EPITHELIUM CELL-SPECIFIC PROMOTER

Publications (1)

Publication Number Publication Date
CN111527102A true CN111527102A (zh) 2020-08-11

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CN201880084798.9A Pending CN111527102A (zh) 2017-11-30 2018-11-28 灵长类动物视网膜色素上皮细胞特异性启动子SynP61

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US (1) US20230193314A1 (https=)
EP (1) EP3717509A1 (https=)
JP (1) JP7390290B2 (https=)
KR (1) KR20200091435A (https=)
CN (1) CN111527102A (https=)
AU (1) AU2018376544B2 (https=)
BR (1) BR112020010775A2 (https=)
CA (1) CA3083404A1 (https=)
CR (1) CR20200235A (https=)
IL (1) IL274885A (https=)
MX (1) MX2020005648A (https=)
PH (1) PH12020550766A1 (https=)
RU (1) RU2020121390A (https=)
SG (1) SG11202005002QA (https=)
WO (1) WO2019106027A1 (https=)

Families Citing this family (13)

* 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
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
US10995344B2 (en) 2015-12-03 2021-05-04 Friedrich Miescher Institute For Biomedical Research SYNP159, a promoter for the specific expression of genes in rod photoreceptors
BR112018011059A2 (pt) 2015-12-03 2018-11-21 Friedrich Miescher Institute For Biomedical Research synp161, um promotor para a expressão específica de genes em fotoreceptores de haste
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
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
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
JP7112414B2 (ja) 2017-02-08 2022-08-03 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ｓｙｎｐ８８
CN111726985B (zh) 2017-11-15 2022-06-10 弗里德里克·米谢尔生物医学研究所 灵长类动物视网膜色素上皮细胞特异性启动子
JP2021503934A (ja) 2017-11-30 2021-02-15 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 網膜色素上皮中の遺伝子の特異的発現のためのプロモーターＳｙｎＰＩＩＩ
JP2022520875A (ja) * 2019-02-25 2022-04-01 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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101426534A (zh) * 2004-02-09 2009-05-06 G·伊南纳 检测和治疗视网膜疾病的方法和组合物
US20170022520A1 (en) * 2014-02-11 2017-01-26 Friedrich Miescher Institute For Biomedical Research Muller cell-specific promoter

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111726985B (zh) * 2017-11-15 2022-06-10 弗里德里克·米谢尔生物医学研究所 灵长类动物视网膜色素上皮细胞特异性启动子

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101426534A (zh) * 2004-02-09 2009-05-06 G·伊南纳 检测和治疗视网膜疾病的方法和组合物
US20170022520A1 (en) * 2014-02-11 2017-01-26 Friedrich Miescher Institute For Biomedical Research Muller cell-specific promoter

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
LAURA FERNANDEZ-SANCHEZ等: "\"Astrocytes and Mtiller Cell Alterations During Retinal Degeneration in a Transgenic Rat Model of Retinitis Pigmentosa", FRONTIERS IN CELLULAR NEUROSCIENCE *
SOO-YOUNG KIM: "Retinal phagocytes in age-related macular degeneration", MACROPHAGE *

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PH12020550766A1 (en) 2021-05-10
AU2018376544A1 (en) 2020-06-11
CR20200235A (es) 2020-10-19
RU2020121390A3 (https=) 2022-02-03
JP2021503933A (ja) 2021-02-15
US20230193314A1 (en) 2023-06-22
JP7390290B2 (ja) 2023-12-01
SG11202005002QA (en) 2020-06-29
MX2020005648A (es) 2020-10-14
BR112020010775A2 (pt) 2020-11-24
AU2018376544B2 (en) 2022-06-16
IL274885A (en) 2020-07-30
WO2019106027A1 (en) 2019-06-06
RU2020121390A (ru) 2021-12-30
KR20200091435A (ko) 2020-07-30
EP3717509A1 (en) 2020-10-07
CA3083404A1 (en) 2019-06-06

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Application publication date: 20200811