CN107531765B - 阴离子通道视紫红质的组合物及其使用方法 - Google Patents

阴离子通道视紫红质的组合物及其使用方法 Download PDF

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CN107531765B
CN107531765B CN201680023358.3A CN201680023358A CN107531765B CN 107531765 B CN107531765 B CN 107531765B CN 201680023358 A CN201680023358 A CN 201680023358A CN 107531765 B CN107531765 B CN 107531765B
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anion
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rhodopsin
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CN107531765A (zh
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约翰·李·斯普迪赫
埃琳娜·G·戈沃鲁诺娃
奥列格·A·西内谢科夫
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University of Texas System
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    • 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
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    • A61N—ELECTROTHERAPY; MAGNETOTHERAPY; RADIATION THERAPY; ULTRASOUND THERAPY
    • A61N5/00—Radiation therapy
    • A61N5/06—Radiation therapy using light
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    • A61N5/062—Photodynamic therapy, i.e. excitation of an agent
    • C—CHEMISTRY; METALLURGY
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • 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
    • C12N15/74—Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora
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    • 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
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    • 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
    • C12N15/82—Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
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    • C12N2510/00—Genetically modified cells

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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN201680023358.3A 2015-03-19 2016-03-18 阴离子通道视紫红质的组合物及其使用方法 Active CN107531765B (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562135470P 2015-03-19 2015-03-19
US62/135,470 2015-03-19
US201562149812P 2015-04-20 2015-04-20
US62/149,812 2015-04-20
US201562261821P 2015-12-01 2015-12-01
US62/261,821 2015-12-01
PCT/US2016/023095 WO2016149599A1 (en) 2015-03-19 2016-03-18 Compositions and methods for use of anion channel rhodopsins

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CN107531765A CN107531765A (zh) 2018-01-02
CN107531765B true CN107531765B (zh) 2021-07-16

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US (1) US10519205B2 (enExample)
EP (1) EP3271376B1 (enExample)
JP (1) JP2018509909A (enExample)
CN (1) CN107531765B (enExample)
AU (1) AU2016232819B2 (enExample)
CA (1) CA2979975A1 (enExample)
WO (1) WO2016149599A1 (enExample)

Families Citing this family (7)

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Publication number Priority date Publication date Assignee Title
JP2018509909A (ja) 2015-03-19 2018-04-12 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム 陰イオンチャネルロドプシンの組成物及び使用方法
US20190367564A1 (en) * 2016-11-17 2019-12-05 The Board Of Regents Of The University Of Texas System Compositions and methods for use of anion channel rhodopsins
CN111518813B (zh) * 2019-02-03 2023-04-28 武汉纽福斯生物科技有限公司 视紫红质的编码序列、其表达载体构建及其应用
US11598792B2 (en) * 2019-05-09 2023-03-07 Howard Hughes Medical Institute Voltage indicators
US20240270799A1 (en) * 2020-03-24 2024-08-15 National University Corporation, Iwate University Modified photoreceptive chloride channel
US20220056100A1 (en) * 2020-08-20 2022-02-24 Board Of Regents Of The University Of Texas System Compositions and methods for use of red-shifted anion channel rhodopsins
JP7756391B2 (ja) * 2021-09-27 2025-10-20 国立大学法人岩手大学 改変光受容クロライドチャネル

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001094384A2 (en) * 2000-06-09 2001-12-13 Fiuotecnica Srl A rhodopsin-like protein extractable from the photoreceptor of euglena gracilis, for use in the preparation of opto-electronic devices
WO2008089003A3 (en) * 2007-01-09 2008-09-25 Univ Leland Stanford Junior System for optical stimulation of target cells

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US992074A (en) * 1910-08-30 1911-05-09 Edgar J Sick Fountain-brush.
US5827702A (en) 1994-10-31 1998-10-27 Genentech, Inc. Ocular gene therapy
IT237434Y1 (it) 1997-04-17 2000-09-13 Philco Italiana Lavatrice con carrello per il suo spostamento
CA2346262A1 (en) 1998-09-17 2000-03-23 University Of Florida Methods for treatment of degenerative retinal diseases
CA2442670A1 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
DE10216005A1 (de) 2002-04-11 2003-10-30 Max Planck Gesellschaft Verwendung von biologischen Photorezeptoren als direkt lichtgesteuerte Ionenkanäle
EP3437473A1 (en) 2006-05-04 2019-02-06 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
JP5322067B2 (ja) 2009-08-10 2013-10-23 国立大学法人東北大学 発現効率が改善された光受容チャネルロドプシン
US20120214188A1 (en) 2010-11-12 2012-08-23 Nathan Klapoetke Light-activated ion channel molecules and uses thereof
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
IL243475B2 (en) 2013-07-09 2023-11-01 Harvard College Multiplex rna-guided genome engineering
JP2018509909A (ja) 2015-03-19 2018-04-12 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム 陰イオンチャネルロドプシンの組成物及び使用方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001094384A2 (en) * 2000-06-09 2001-12-13 Fiuotecnica Srl A rhodopsin-like protein extractable from the photoreceptor of euglena gracilis, for use in the preparation of opto-electronic devices
WO2008089003A3 (en) * 2007-01-09 2008-09-25 Univ Leland Stanford Junior System for optical stimulation of target cells

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
Algal nuclear genomes reveal evolutionary mosaicism and fate of nucleomorphs;Curtis等;《NATURE》;20121206;第492卷(第7427期);第59-65页 *
Curtis等.Algal nuclear genomes reveal evolutionary mosaicism and fate of nucleomorphs.《NATURE》.2012,第492卷(第7427期),第59-65页. *
Independent optical excitation of distinct neural populations;Klapoetke等;《Nature Methods》;20140209;第11卷(第3期);第338-346页 *
Klapoetke等.Independent optical excitation of distinct neural populations.《Nature Methods》.2014,第11卷(第3期),Independent optical excitation of distinct neural populations. *
Structure-Guided Transformation of Channelrhodopsin into a Light-Activated Chloride Channel;BERNDT等;《Science》;20140425;第344卷(第6182期);第420-424页 *

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Publication number Publication date
CA2979975A1 (en) 2016-09-22
WO2016149599A1 (en) 2016-09-22
US20180118793A1 (en) 2018-05-03
EP3271376A1 (en) 2018-01-24
CN107531765A (zh) 2018-01-02
EP3271376B1 (en) 2022-05-25
AU2016232819B2 (en) 2021-10-21
US10519205B2 (en) 2019-12-31
JP2018509909A (ja) 2018-04-12
AU2016232819A1 (en) 2017-10-05
EP3271376A4 (en) 2018-11-07

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