JP2020501538A - インターニューロン中の遺伝子の特異的発現のためのプロモーターsynpi - Google Patents

インターニューロン中の遺伝子の特異的発現のためのプロモーターsynpi Download PDF

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JP2020501538A
JP2020501538A JP2019529228A JP2019529228A JP2020501538A JP 2020501538 A JP2020501538 A JP 2020501538A JP 2019529228 A JP2019529228 A JP 2019529228A JP 2019529228 A JP2019529228 A JP 2019529228A JP 2020501538 A JP2020501538 A JP 2020501538A
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nucleic acid
cell
gene
sequence
acid sequence
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JP2020501538A5 (enExample
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ロスカ,ボトンド
ユエットナー,ジョゼフィーヌ
テイシェイラ,ミゲル
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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
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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Publication of JP2020501538A publication Critical patent/JP2020501538A/ja
Publication of JP2020501538A5 publication Critical patent/JP2020501538A5/ja
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
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  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2019529228A 2016-12-01 2017-11-29 インターニューロン中の遺伝子の特異的発現のためのプロモーターsynpi Pending JP2020501538A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022002979A JP2022050590A (ja) 2016-12-01 2022-01-12 インターニューロン中の遺伝子の特異的発現のためのプロモーターsynpi

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16201749.5 2016-12-01
EP16201749 2016-12-01
PCT/EP2017/080829 WO2018099975A1 (en) 2016-12-01 2017-11-29 Synpi, a promoter for the specific expression of genes in interneurons

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JP2022002979A Division JP2022050590A (ja) 2016-12-01 2022-01-12 インターニューロン中の遺伝子の特異的発現のためのプロモーターsynpi

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JP2020501538A true JP2020501538A (ja) 2020-01-23
JP2020501538A5 JP2020501538A5 (enExample) 2021-01-14

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JP2019529228A Pending JP2020501538A (ja) 2016-12-01 2017-11-29 インターニューロン中の遺伝子の特異的発現のためのプロモーターsynpi
JP2022002979A Withdrawn JP2022050590A (ja) 2016-12-01 2022-01-12 インターニューロン中の遺伝子の特異的発現のためのプロモーターsynpi

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US (1) US20190376083A1 (enExample)
EP (1) EP3548094A1 (enExample)
JP (2) JP2020501538A (enExample)
CN (1) CN110072558A (enExample)
WO (1) WO2018099975A1 (enExample)

Families Citing this family (12)

* 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
US10941417B2 (en) 2015-04-30 2021-03-09 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in Müller cells
ES2926677T3 (es) 2015-09-15 2022-10-27 Friedrich Miescher Institute For Biomedical Res Herramientas y métodos terapéuticos novedosos para tratar la ceguera mediante el direccionamiento a los fotorreceptores
US11254934B2 (en) 2015-10-14 2022-02-22 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in retinal endothelial cells
US10994026B2 (en) 2015-12-03 2021-05-04 Friedrich Miescher Institute For Biomedical Research SynP160, a promoter for the specific expression of genes in rod photoreceptors
CA3006952A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp161, a promoter for the specific expression of genes in rod photoreceptors
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
WO2017093931A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp159, a promoter for the specific expression of genes in rod photoreceptors
AU2017354086C1 (en) 2016-11-02 2021-05-06 Friedrich Miescher Institute For Biomedical Research SynP198, a promoter for the specific expression of genes in direction selective retinal ganglion cells
US11371060B2 (en) 2017-02-08 2022-06-28 Friedrich Miescher Institute For Biomedical Research SYNP88, a promoter for the specific expression of genes in retinal ganglion cells
CR20200210A (es) 2017-11-15 2020-09-23 Friedrich Miescher Institute For Biomedical Res Promotor especìfico de las cèlulas del epitelio pigmentario retinal en primates
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006519584A (ja) * 2002-10-31 2006-08-31 ジヤンセン・フアーマシユーチカ・ナームローゼ・フエンノートシヤツプ Cnsにおけるcrh応答性遺伝子
WO2013068413A1 (en) * 2011-11-08 2013-05-16 Novartis Forschungsstiftung, Zweigniederlassung, Friedrich Miescher Institute For Biomedical Research Rod cell-specific promoter
US20130195763A1 (en) * 2012-01-27 2013-08-01 Charles F. Landry Methods and compositions for improving sleep and memory

Family Cites Families (2)

* 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
US20110098346A1 (en) * 2008-05-19 2011-04-28 Agency For Science, Technology And Research Nucleic acid molecule and method of targeting gene expression to gliomas

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006519584A (ja) * 2002-10-31 2006-08-31 ジヤンセン・フアーマシユーチカ・ナームローゼ・フエンノートシヤツプ Cnsにおけるcrh応答性遺伝子
WO2013068413A1 (en) * 2011-11-08 2013-05-16 Novartis Forschungsstiftung, Zweigniederlassung, Friedrich Miescher Institute For Biomedical Research Rod cell-specific promoter
US20130195763A1 (en) * 2012-01-27 2013-08-01 Charles F. Landry Methods and compositions for improving sleep and memory

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
DEVELOPMENT, vol. 121, no. 6, JPN6021039832, June 1995 (1995-06-01), pages 1719 - 1730, ISSN: 0004760202 *
NEUROSCI RES., vol. 68, no. 2, JPN6021039836, October 2010 (2010-10-01), pages 94 - 102, ISSN: 0004760203 *

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CN110072558A (zh) 2019-07-30
EP3548094A1 (en) 2019-10-09
WO2018099975A1 (en) 2018-06-07
JP2022050590A (ja) 2022-03-30
US20190376083A1 (en) 2019-12-12

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