CN101484005A - 通过向体内递送视紫红质核酸恢复视觉响应 - Google Patents

通过向体内递送视紫红质核酸恢复视觉响应 Download PDF

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CN101484005A
CN101484005A CNA2007800252180A CN200780025218A CN101484005A CN 101484005 A CN101484005 A CN 101484005A CN A2007800252180 A CNA2007800252180 A CN A2007800252180A CN 200780025218 A CN200780025218 A CN 200780025218A CN 101484005 A CN101484005 A CN 101484005A
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promotor
retina
sequence
retinal
light
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潘卓华
亚历山大·M·季泽尔
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Wayne State University
Pennsylvania College of Optometry
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Wayne State University
Pennsylvania College of Optometry
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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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
    • C12N15/85—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
    • A61K38/00—Medicinal preparations containing peptides
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CNA2007800252180A 2006-05-04 2007-05-04 通过向体内递送视紫红质核酸恢复视觉响应 Pending CN101484005A (zh)

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US79735706P 2006-05-04 2006-05-04
US60/797,357 2006-05-04

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CN101484005A true CN101484005A (zh) 2009-07-15

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US (4) US8470790B2 (https=)
EP (2) EP2019588A4 (https=)
JP (5) JP2009536219A (https=)
CN (1) CN101484005A (https=)
AU (2) AU2007247929A1 (https=)
CA (1) CA2685900A1 (https=)
WO (1) WO2007131180A2 (https=)

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CN101693127B (zh) * 2009-09-30 2014-10-08 深圳先进技术研究院 皮肤材料及其制备方法
CN107012171A (zh) * 2011-04-22 2017-08-04 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
CN107530449A (zh) * 2015-02-23 2018-01-02 Ucl商业有限公司 改善视觉的基因疗法
CN109661471A (zh) * 2015-12-04 2019-04-19 索邦大学 启动子及其用途
CN109716198A (zh) * 2016-06-17 2019-05-03 索邦大学 以可控的光强度照射物体的装置及相关方法
CN110072557A (zh) * 2016-11-02 2019-07-30 弗里德里克·米谢尔生物医学研究所 用于在方向选择性视网膜神经节细胞中特异性表达基因的启动子SynP198
CN110267673A (zh) * 2016-04-29 2019-09-20 珍视生物股份公司 利用chrimson来进行光遗传视觉恢复
CN110521842A (zh) * 2019-05-06 2019-12-03 高思 人眼晶状体和视网膜感光细胞营养素裹养眼明目草药糖果
CN111032068A (zh) * 2017-04-12 2020-04-17 马克斯·普朗克科学促进学会 新的光遗传学工具
US10883117B2 (en) 2015-03-24 2021-01-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
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US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
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