KR20200091435A - 영장류 망막 색소 상피 세포-특이적 프로모터 SynP61 - Google Patents

영장류 망막 색소 상피 세포-특이적 프로모터 SynP61 Download PDF

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Publication number
KR20200091435A
KR20200091435A KR1020207018368A KR20207018368A KR20200091435A KR 20200091435 A KR20200091435 A KR 20200091435A KR 1020207018368 A KR1020207018368 A KR 1020207018368A KR 20207018368 A KR20207018368 A KR 20207018368A KR 20200091435 A KR20200091435 A KR 20200091435A
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nucleic acid
acid molecule
acid sequence
retinal pigment
isolated nucleic
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Korean (ko)
Inventor
요제피네 위트너
야체크 크롤
보톤트 로스카
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프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치
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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)
KR1020207018368A 2017-11-30 2018-11-28 영장류 망막 색소 상피 세포-특이적 프로모터 SynP61 Ceased KR20200091435A (ko)

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
KR20200091435A true KR20200091435A (ko) 2020-07-30

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Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020207018368A Ceased KR20200091435A (ko) 2017-11-30 2018-11-28 영장류 망막 색소 상피 세포-특이적 프로모터 SynP61

Country Status (15)

Country Link
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

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7309487B2 (en) * 2004-02-09 2007-12-18 George Inana Methods and compositions for detecting and treating retinal diseases
WO2015121793A1 (en) * 2014-02-11 2015-08-20 Friedrich Miescher Institute For Biomedical Research Müller cell-specific promoter
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

Also Published As

Publication number Publication date
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
CN111527102A (zh) 2020-08-11
IL274885A (en) 2020-07-30
WO2019106027A1 (en) 2019-06-06
RU2020121390A (ru) 2021-12-30
EP3717509A1 (en) 2020-10-07
CA3083404A1 (en) 2019-06-06

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