JP2022512784A - 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ＳｙｎＰ７８（ＰｒｏＡ２７） - Google Patents

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ＳｙｎＰ７８（ＰｒｏＡ２７） Download PDF

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JP2022512784A
JP2022512784A JP2021521854A JP2021521854A JP2022512784A JP 2022512784 A JP2022512784 A JP 2022512784A JP 2021521854 A JP2021521854 A JP 2021521854A JP 2021521854 A JP2021521854 A JP 2021521854A JP 2022512784 A JP2022512784 A JP 2022512784A
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nucleic acid
gene
cell
expression cassette
retinal ganglion
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ユエットナー，ジョゼフィーヌ
クロル，ヤツェク
ロスカ，ボトンド
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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
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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
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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)
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  • Genetics & Genomics (AREA)
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  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2021521854A 2018-10-25 2019-10-24 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ＳｙｎＰ７８（ＰｒｏＡ２７） Withdrawn JP2022512784A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18202537 2018-10-25
EP18202537.9 2018-10-25
PCT/IB2019/059091 WO2020084540A1 (en) 2018-10-25 2019-10-24 Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells

Publications (2)

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JP2022512784A true JP2022512784A (ja) 2022-02-07
JP2022512784A5 JP2022512784A5 (https=) 2022-10-31

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JP2021521854A Withdrawn JP2022512784A (ja) 2018-10-25 2019-10-24 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ＳｙｎＰ７８（ＰｒｏＡ２７）

Country Status (5)

Country Link
US (1) US20210355505A1 (https=)
EP (1) EP3870240A1 (https=)
JP (1) JP2022512784A (https=)
CN (1) CN112930201A (https=)
WO (1) WO2020084540A1 (https=)

Families Citing this family (12)

* 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.
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
EP3383440B1 (en) 2015-12-03 2021-06-30 Friedrich Miescher Institute for Biomedical Research Synp162, 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
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
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
RU2766353C2 (ru) 2016-11-02 2022-03-15 Фридрих Мишер Инститьют Фор Байомедикал Рисерч Synp198, промотор для специфической экспрессии генов в ганглионарных клетках сетчатки, избирательных в отношении направления
WO2018146588A1 (en) 2017-02-08 2018-08-16 Friedrich Miescher Institute For Biomedical Research Synp88, a promoter for the specific expression of genes in retinal ganglion cells
MX2020005033A (es) 2017-11-15 2020-10-28 Friedrich Miescher Institute For Biomedical Res Promotor especifico de las celulas del epitelio pigmentario retinal en primates.
SG11202005024QA (en) 2017-11-30 2020-06-29 Friedrich Miescher Institute For Biomedical Res Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002082904A2 (en) * 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
US9567376B2 (en) * 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
EP3383440B1 (en) * 2015-12-03 2021-06-30 Friedrich Miescher Institute for Biomedical Research Synp162, a promoter for the specific expression of genes in rod photoreceptors
EP3176177A1 (en) * 2015-12-03 2017-06-07 Friedrich Miescher Institute for Biomedical Research Synp157, a promoter for the specific expression of genes in rod photoreceptors

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Publication number Publication date
US20210355505A1 (en) 2021-11-18
CN112930201A (zh) 2021-06-08
EP3870240A1 (en) 2021-09-01
WO2020084540A1 (en) 2020-04-30

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