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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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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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
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    • 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)
  • Biomedical Technology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
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  • Immunology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (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

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

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2010011467A (es) 2008-04-18 2011-02-15 Novartis Forschungsstiftung Novedosas herramientas y metodos terapeuticos para tratar la ceguera.
EP3289090B1 (en) 2015-04-30 2019-06-26 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
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
CN108474001B (zh) 2015-12-03 2022-04-08 弗里德里克·米谢尔生物医学研究所 SynP160，用于基因在视杆光感受器中特异性表达的启动子
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
ES2761328T3 (es) 2015-12-03 2020-05-19 Friedrich Miescher Institute For Biomedical Res SynP161, un promotor para la expresión específica de genes en fotorreceptores de bastón
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
SG11202004205YA (en) 2017-11-15 2020-06-29 Friedrich Miescher Institute For Biomedical Res Primate retinal pigment epithelium cell-specific promoter
AU2018376259A1 (en) 2017-11-30 2020-06-11 Friedrich Miescher Institute For Biomedical Research SynPIII, a promoter for the specific expression of genes in retinal pigment epithelium
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
SG11202004205YA (en) * 2017-11-15 2020-06-29 Friedrich Miescher Institute For Biomedical Res Primate retinal pigment epithelium cell-specific promoter

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

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Application publication date: 20200811